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Teams for the talk today. One of the things to think about, I think in a career as a future is the role of A I in any career including in neurology as we move forward. Um This is what I was told to talk about about me basically and then about the field. So I will try and cover all all of these things to give you an idea, I guess about number one, how you become a neurologist? And I guess secondly, about the field itself, what I find interesting about neurology. Um So where do I work? Is actually a slightly confident I work in uh six different places I worked out in this line together. Um uh Overall, I would say I work at the Queen Square Center for pneum Muscular Diseases, we on the uh the clinical lead uh there. But actually that is a not a virtual center, but actually it's a center which is half NHS in terms of the National Hospital for Neurology Neurosurgery, which is where my contracts actually held. Uh But a lot of research also is done there, which is through University called London Institute of Neurology. So I have both uh research funded time as well as predominantly uh uh NHS funded time. As mentioned, I also work one of my roles rather as as the undergraduate lead of education for medical students who come to Queen Square. So that's through the UCL medical school. I spent a day a week. In fact, it was today on Wednesdays at a district general Hospital doing neurology list, the hospital in Stevenage. And I also do work at the main hospital in the trust which is a university called hospital. So I do have a very busy time and a very job um timetable for the week. This is roughly how my official timetable is split. 30% of my time is academic, 40% is clinical through the Queen Square Hospital. 15% is clinical through UC H. 15% of my time officially is assigned to the medical students and 25% of my time is assigned to the D th listed hospital. And I'm sure or you smart medical students in the audience will realize that that adds up to more than 100%. And that certainly is how it feels. On the other hand, it is a really nice way to spend a week today. For example, I spend the entire day at this hospital in Stevenage. My mom there now one day a week to start with, I was there three days a week. But it does give me a real genu experience where I see everything and anything in neurology, because obviously my time with university is highly specialized as I'll discuss. But, you know, I think it really is opportunity in neurology and to be fair, all the medical specialties to have a very job plan and to keep yourself very interested. So now I'm going to move on to how I got to where I did. And again, obviously everybody's story is different and none of you will start where I started, which was at the University of Auckland in New Zealand. But at the same time, I think there's some interesting lessons here. And again, I'll discuss that as I go through as well as at the end. So the University of Auckland, in fact, I grew up in Wellington in New Zealand. Um and being not from a medical family, as far as I was aware, there was only two medical schools in New Zealand. One was University of Auckland and the other was in the University of Otago and the advanced University of Auckland was that you got straight in from high school where it University of Otago, you had to do a premed year and then apply. So when I got in straight from high school, that was the obvious university to go to back then. It was again structured a lot like some UK universities, three preclinical years at the medical school. And at the end of my three preclinical years was the last long holiday. So I actually came to London and worked in a pub called Pinder Rose Oak. And we'll come back to that later on. However, I came back and then did three years clinical training in the various hospitals around Auckland. This is our Auckland Hospital itself. Um And at the end of that, of course, I guess when you come to the end of your medical training, the first question always is, where do you want to work for your first house job there? Always we to call them back then house officer jobs. And again, I thought about this quite hard and I decided that the best place to work actually for your first house job is number one, a nice place to live. So I lived in a lovely part of New Zealand called the Hawke's Bay, which has some of the best weather in New Zealand. But also, again, it was very much a district general hospital that served a population of a couple of 100,000 people, a quarter million people. And it was a really great place to work. And I think one of the lessons I'd like to say is even if you want to be the most specialized neurologist in the end, it is always best to start with a very broad base and then specialize from there because without that broad understanding of medicine of neurology, etcetera, it's very hard to be very good at something very specialized. So again, that's one thing I would encourage from there. After a couple of years, I actually went and worked in Scotland and Glasgow for various reasons. So I came back to the UK and did a year there. At the end of my time there, I got a job as a registrar. I was what we call a junior registrar back in New Zealand, actually back in my hometown of Wellington. And I remember distinctly having achieved this job and I was kind of, I think I was traveling at the time I traveled on the way back and being asked you what specialties or what runs I wanted to do when I went back to New Zealand or when I started the job. And interestingly, as a medical student, I never did neurology. It was a medical specialty you had to choose. And I asked for neurology and didn't get it. I did renal medicine and respiratory. I hadn't done any neurology placement and I still knew somehow, which is interesting that neurology I always found really interesting again, working obviously at ad GH in general medicine, you would see all the different patients and the nodule ones always interesting. So when I was asked in Scotland, when I came back to Wellington, what I wanted to do, I said, I'd love to do neurology and I said, oh, ok. Yeah, that's great. Actually, there's plenty of opportunities in neurology in Wellington and at the time, the neurology department in Wellington had a kind of a reputation for being a hard place to work, neurologists. Some of us are interesting. People put it that way. And I think the record was a registrar lasted four days before asking to change because I didn't know any of this. And I said, no, I wanted Gener. That's great. So I came back to Wellington. In fact, my first run as a registrar in New Zealand was neurology and it was the most amazing experience because I did eight clinics a week as well as seeing all the ward patients for referrals, et cetera. So I learned so much in that first six months and then moved on. As I said, the New Zealand system still very much did a lot of general medicine as well. So I finished my general medicine training and started my advanced training in neurology in New Zealand. The next kind of pivotal moment really in my progression was one of those random things. So I was still a year and a half from finishing my training. And I went to a conference in Australia near Ayers Rock, Uluru, the Australia New Zealand Association Rogers meeting. And at that conference, it turned out that there were fellowships available to the UK actually to the UK as well as to the Mayo Clinic in America. And um at that time, they'd actually already closed and applications had happened. But for some reason that year, there weren't enough applicants. So they said at this meeting, if anybody else wants to apply, you can. And again, one of my consultants in Wellington actually had worked at Queen's Square and said, look, you really must apply. And I said I'm too junior. There's no chance I'll get it. But at his insistence, I put in an application and fortuitously that's Queen Square and the blur there, it was just a blur in my eyes at the time I got the post. So my final clinical year of training was to be as a registrar at the National Hospital for neurosurgery. Which to be honest, prior to that meeting, I had never even heard of what I actually did in the end was I had to do my next run in New Zealand, which was in Auckland. So back across to Auckland and then finally in 2008, ah, came and acted as a registrar at Queens Square for my final year. Interestingly, and again, I had absolutely no idea about this when I worked at PSE Oak, but it was about a five minute walk from the pub. I worked in 10 years prior. So I'd kind of ended up working just 10 minutes away from where I worked all that time ago as a bartender. Um, and so we not necessarily pso there's many of you who have been to Queens Square, there's a pub right on Queen's Square called the Queens Lader. That's where on my very first day. There I met who was to become my phd supervisor. So, um and so yeah, definitely in association with Queen Square. Um And so yes, at the end of or actually during my training, my one year fellowship at Queen Square as a registrar, I knew I wanted to do research. So as I approached one of the professors there in New Ma Disease and asked Professor Riley what options there were. And luckily there was, they just set up a new research center and had a number of fellowships that they could effectively directly award. They had the money, it was an interview, et cetera, but they had the funding already to do that. And hence I started a phd through UCL Institute of Neurology P is the most ugly building in London on the corner of Queen Square, right beside that beautiful old urology hospital building. In fact, they're about to knock that down. There's plans to knock it down. We're halfway through building a brand new Neurology institute a few minutes walk away. But yes, I started my phd in 2009, the job that was going or one of the jobs that was going that seemed most interesting was in muscle MRI. So I spent the next four years and I'll talk about that a little bit later on learning all about muscle MRI and including my phd at the end of that. And again, an extra year doing more research, I got my consultant kind of credentials. And again, much like when I first started at medical school, I really thought that the best thing to do to start with is to keep things general. And so I got a half time job working as a neurologist and it is now 2013, at least hospital in Stevenage. Again, this is something I would really encourage you all regardless of where you're going into. One could do the option where one immediately does new muscle disease at Queen Square. But I think again, it makes you a bit of a neurologist. It makes you more broadly flexible, but also, um it makes you better neoma neurologist knowing about other aspects. I mean, the the worst thing in the world would be if, you know, I had a patient who had a tremor, I couldn't, you know, assess them even if I was seeing them at the neurology hospital. So um I started working there half time and continued some research and work at Queen Square at the same time. And that brings me on to now, really my role at the moment is in those three places. As I mentioned, I still have an academic role. I supervise four phd students. Um for example, through Queen's Square, as well as the medical students, et cetera. I run a master's course as well. I have the role at Steve. It has gone down gradually over the years and it now sets down to one day a week. I also have a major role in at Queen Square itself as a clinical lead in your master disease. Of course, there's still well pin oak but Queens louder on the corner, there's the social side as well. And I still find time to fly home to go to New Zealand as well, which is important. And so, yes, my time has been New Zealand and the UK, mainly with that kind of key time in Australia which link the two. So again, as I've said through the story, I think the lessons I've learned, which I would apply to neurology, but again, would apply to area of medicine you're doing is to always start general and then specialize the advantage of that. Of course, is that you um I guess find out what you enjoy and then do what you enjoy. Again in the UK, it used to be the case that to get a training number in urology, you more or less needed a phd. So you would finish your sho jobs and then you would go and do a phd and then you would start your advanced training where either the other way around, I picked my phd at the end of my training. And of course, the advantage of that was I knew what I was interested in and therefore I could just continue on afterwards where there are people who do a phd and then end up in some other area because they discover they like something else. So again, that's an advantage to that, but that applies at all stages of your career. The second thing I would really encourage and of course, you'd say I would say that is to travel again, you don't have to travel halfway around the world. But I do think traveling and working in a completely different health service is an even more broadening experience. But just, you know, working in different places around the UK, seeing how different things are done, having jobs in different places, experiencing different things, really does broaden your experience and make you a better doctor at the end of it. And the third thing I think that we learn from this is that you always take those opportunities as they arise when something's come up, whether it was the phd opportunity opportunity to come to London, you know, you have, you have to take the opportunity, you're never quite sure when they're going to arise. But again, if you make the most of the opportunities, you'll never know where that might lead you. All right. So enough about uh well, it's still about me, but now moving on to neurology, what do I enjoy about the field? What is difficult? Well, in terms of what I enjoy, I think the thing that really drew me to neurology and again, hopefully how other doctors see neurologists is you do need extremely good diagnostic skills in neurology, you know, the thing that drew me to medicine really is the problem solving aspect. And neurology is really about taking a patient and working out exactly where the problem is localizing and what the problem is. And that really is the basic skills of medicine being history, taking skills and examination skills. Yes, we have fancy tests. But if you don't get those basics right, you'll just lead yourself down a garden path. And so, you know, this kind of leads on to the next topic really is that the reason that diagnosis, schools are so important is because of the diagnostic diversity in neurology, we'll talk about that in a second. But you know, if you look at my general neurology clinic in Stevenage, there isn't a diagnosis that more than 10% of the new patient referrals have. Um I think they did a survey on this and roughly 10% of referrals, a headache. But you know, there really is a million different diagnoses in neurology and that's why those diagnostic skills are so important. And again, in some ways, you can look up the knowledge you can work out a localization, a type of disease. And then you can look up the details of it or what tests you should do or you can obviously look up the best treatments. Um of course, as you become experienced with the disease, you know, these, but if it's a disease you haven't heard of and again, you'll be surprised but still 10 years as a neurologist every month, at least I see a patient with some syndrome or eponymous thing that I haven't heard of before or a new gene or et cetera, et people are so exciting and yet the process is the same to get there. And that's where those skills are so important, I think as well. This really is why medicine will remain a specialty because yes, you know, if you can feed the algorithm all the right information, it probably can make a diagnosis. But actually getting that right information is the skill. Obviously, the examination skills are something that computers won't be able to regenerate in the in the near future. And finally, of course, um the the skills that are not just neurology would come across all the medicine, compassion, kindness, empathy, these kind of things at the moment. Again, I think even if a computer was mi them, well, it wouldn't feel the same as when a doctor does. So again, I think you guys have still a long career ahead of you. The thing that's really exciting about neurology now and I'll talk about this at the end is that we in neurology are at the forefront of gene therapy advances. So just in my clinic from five years ago, we had no gene therapies, we now have six different gene therapies that we're giving. And again, the last 20 years have been about the developments and genetic testing and knowledge about genes that's expanded to the point that we can now diagnose most patients with genetic diseases, we can find the gene in most cases. And it does feel like we're at that kind of uptick in the directory of gene treatments. I'll talk about that in a bit, but that's extremely exciting to go from the field where, you know, you had these great dial at schools but no treatments to ones where we have amazing treatments. And of course, the treatments in MS have come in the last 20 years and, and that's a completely different disease to what it was when I started my career in neurology. And again, we're moving that way with genetic diseases. The final thing I do enjoy about my specialty is weekends and I don't do any weekend on call. Again, there are opportunities to do that if you wish. But even though it's fairly light. Um uh and again, versus other specialties, it is roughly speaking, uh uh a Monday to Friday job, what's typical about the official team. Um I think still, although, you know, as I say, the gene therapies are advancing, there still is this kind of view or a neurologist that they make amazing diagnosis, you have motor neuron disease. And I can tell that as just by talking to you and examining you and then while there's not much we can do about it. Unfortunately, again, obviously, that's a flippant way of looking at things and the reality is there's a lot that doctors do, even if the disease is in a comma is untreatable. A lot, a lot of benefit we give to our patients. Um, and even in an untreatable disease in a bit comma like Duchenne muscular dystrophy for which we're just starting to get a couple of gene therapies for the life expectancy in that condition has gone from 18 to over 40 in the last 2025 years without in the V comma's gene treatment. But with just amazing supportive care. So there's a lot that we do do beyond cures. And the other thing that again is difficult and this applies to all specialties is the NHS pressures working in the NHS. And again, although I enjoy my weekends, I don't enjoy my evenings so much and I do work hard during the week and don't often get home on what we've considered on time. And again, it's to do with that job plan I gave you, I do work 100 and 20% of a week according to my job plan and that extra 20% is done by working harder during the week and I keep my weekends free and don't get me wrong. You don't have to do that. Um I have a very good colleague in Stevenage who just works in Stevenage. He arrives at eight and he leaves at four each day, Monday to Friday. Um, so again, it depends on what job you take on. Um But yes, I the pushes apply to all of us hobbies and projects. I thought, I thought this was quite interesting actually because when I think about what I am, which as I say is an expert in quantitative MRI at the bottom, which we can talk about. There's actually quite a few steps from being a doctor down to that point. And the other interesting thing is that, um you know, when people ask me why I became a neurologist or a neuromuscular specialist or a muscle specialist or a quantitative MRI specialist. Really a lot of it is about these kind of individual decisions that you take because you don't start off or I didn't think I want to be a muscle MRI specialist. First of all, actually, I had to decide I want to be a doctor because of being an engineer, I was almost an engineer. And then, in fact, when I entered medical school, I thought I wanted to be a psychiatrist, which interestingly is not that far from being a neurologist. When you think about it, it's a different college in the UK. But in America, the neurologists are part of the College of Neurologists and psychiatrists because it's the same organ we deal with the brain and in some ways what happens, I think and again, everybody's different. Some people know they want to be a hand surgeon or something very specific, you know, from the start of medical school and maintain that. But I think more commonly, you kind of get, you have a feeling but you kind of work out what you don't want to do. So, very early on, for lots of reasons. I knew I didn't want to be a surgeon. And then as I say, as I was doing the medical specialties in my training, neurology was most interesting to me. The second most interesting specialty to me was hematology. You might say that's just because of my name. And oddly enough, now I get called Dr Marrow quite frequently, but I was actually doing a six month hematology run. Um which I found very interesting, actually, very high pressure because we were on a, a bone marrow transplant unit. And these are patients who are either cured and they're young and healthy or they die. And that was, you know, really, really hard. Um But again, what I discovered on the unit is on my six month of hematology is that the patient I found most interesting was someone who had itp, had low platelets, but they developed this odd kind of thing where they knocked off their nerve, they got a radial nerve palsy and then a median nerve palsy. So at that point, I discovered that probably I found neurology more interested in the hematology. And then again, within neurology, as we'll discuss, there are different specialties. How do they come to nec disease? I think the neuromuscle disease specialist is the ultimate diagnostician because of, you know, how important the examination is and then into muscle specialists. And again, of course, you can stop at any of these points. As I say, my colleague in Steven Edge is primarily a gen neurologist. He's an interest in movement disorders. So he does some but next and see some of the Parkinson's patients, but 80% of the time his time is spent doing general neurology And so you can decide how far down this line to come. And ultimately, what I'm interested in now is neuromuscular MRI. And what we do is we take quantitative images of people's legs and measure the exact amount of fat and water and other things in them. And then use this to show how well it correlates with the patient's function and ultimately show how it progresses with time and correlates and measure the disease progression. And therefore we're now using these techniques that I developed or help develop to be fair ah in clinical trials to show that these gene therapies have been advanced by other clever scientists will be beneficial in our patients. So again, right down at the end, one thing we're moving on to very recently is using A I segmentation to speed up the process and make the analysis more accurate. So again, right from that first point where I was at high school thinking, should I become a doctor or not? You know, again, that series of choices and things in your mind lead you down a certain path in a career that I really like. So again, I guess my message here is don't be too worried if at the moment, you're not sure what kind of doctor you want to be, it will become apparent with time. And the other great thing about medicine is that, you know, it really isn't one job. It's a million different jobs. You know, if you discover, again, this is not me, obviously, if you discover you don't really like talking to patients, then, you know, you can become a, a radiologist or a pathologist or, or a researcher or academic or an, or um, epidemiologist. You know, they really are a public health ps position. There really are so many areas that, you know, it's not just one job, it's a vocation and then you can find the area that suits your skills the best, you know, personally at school. I was always best at math. So I've kind of gone into quite a kind of a mathematical field. But again, you can take medicine whichever way you like. This is a I's interpretation of neuromuscular MRI. Um, I do have other hobbies. I wasn't sure I was meant to talk about these. This is a neurologist riding a green bike. This is me at the weekends usually. And again, I think it really is important to have time, not just for your career, but I think throughout your career. It's a medical school, junior doctor or as a consultant to do things outside of medicine. So I have to be honest, outside of medicine, my two things are family and cycling. But again, I won't talk too much more about that unless you wish me to um about the field was the next section we doing ok for time. So as I say, in terms of special interests, there really is an amazing opportunity, neurology to specialize in something. And again, these are only the first level of specialization. So headache, obviously a major specialty neuromuscular, which is a nerve and muscle disease. But again, as a good example, at Queen's Square, we don't just have neuromuscular, we have the muscle clinic, the nerve clinic, the myotonia clinic and the motor neurone disease slack. And within the muscle clinic, there's general muscle, there's mitochondrial disease, there's transition muscle, there's metabolic muscle. So again, there's highly specialized things with each of these areas. The two stars, there are interesting because there are two areas which are clearly part of neurology. These are diseases of the brain and yet for various reasons, mainly commissioning and workforce, they've moved away from neurology somewhat. So stroke was used to be looked after by general medicine specialists or geriatricians. It's now especially in its own right. So you can dual train in stroke and neurology or you can dual train in stroke in general medicine without doing neurology. And coch neology is really interesting as well because again, people are often surprise at this, but the commissioning the money for diagnosed people with dementia in the UK is given to, again, old age psychiatrists. It's not that neurologists don't want to do it. And again, there is co neurology as a specialty, but it's just that we don't simply have a number of neurologists to be able to provide that. Well, interestingly again, you've probably seen this in the news this year. There are now disease modifying therapies for Alzheimer's. So it's kind of become like MS was 20 years ago. And again, psychiatrists are not trained to give dangerous treatments. There's not even a place to give them within, within their hospitals, within their units. So they have to be a complete rethinking cog neurology and either upskilling and changing the way psychiatrists work or integrating neurologists again, in the care more, that's going to be a major change over the next 10 years, common cases. So again, these really reflect the list on the previous page if you look at my follow up patients and again, I should say as well, I haven't actually said this, but neurology is predominantly an outpatient specialty. So yes, at Queen Square, obviously, we have massive neurology awards but at in fact, only Neurology Awards and neurosurgery of all types, but actually at Lister and Steven, if there are Neurology awards, patients who need to be admitted, come under General Medicine and then we consult on them. But you know, most of our time is spent seeing outpatients with chronic conditions. One of the probably the commonest reason for a referral is migraine headache generally, but specifically migraine, which is again, a fascinating area which recently has got a new and really exciting treatment CG RP antagonists. So that's a lot of the initial patients. But again, they make up far less of the follow up patients because one would advise treat and then discharge back to the GP epilepsy. And seizure disorder is obviously very common movement disorders, particularly Parkinson's disease, new inflammation or for sclerosis. New is interesting because as a field, as big as MS and Parkinson's and epilepsy, but really combines a lot of really interesting diseases. The my tooth disease was one of the diseases I did my phd in that's an inherited opathy, but near myasthenia gravis and motor neuron disease. The other really common conditions in that field CMT actually is the commonest inherited mental disorder. One in 2.5 1000 people have CMT really common. And again, it really crosses across all these different areas of function disorders. So again, this is where really we get the interface between psychiatry and neurology. People who have, for example, non epileptic seizures or functional weakness or or functional tremor, functional blindness. If you name a symptom, you can have it as a functional disorder. Again, it's really important to understand those orders because again, the right individual at the right time can really make a difference. Again, more generally in terms of disorders, stroke and dementia. But again, for a reason, we've discussed, I don't see those patients often in my clinic. Although dementia in particular, we still see patients with young onset dementia or interesting dementia so to speak, they asked me to mention pay potential. And so this is a really straightforward in the sense that as an NHS consultant, you receive the NHS consultant pay scale, which again has gone up recently. Again, I'm not going to talk about strikes and things, but roughly speaking, it starts off at 100,000 as a consultant and goes up to 100 and 30,000 as a senior consultant. If you were 100 and 20% of the week, you get slightly more. I don't personally do any private work at all for a lot of reason, money, I'm too busy, but obviously in neurology or in neurology, there is definitely the potential for private work. A lot of my colleagues at Queen Square do the thing where you have 80% of your time doing NHS work and 20% doing private work or something like that. There aren't many neurologists who only work in private and private obviously pays significantly more than public. Again, I can't tell you how much because I don't do it, but it does also come with its own challenges. It's fair to say I see a lot of ex private patients who come to the NHS. Right. The final thing I want to talk about the next 10 minutes. And again, this really is the most exciting thing I, I've eluded with this a number of times. I don't know how many of, you know, this uh story of this treatment. But really, it is something that has, is, is, is life changing for the patients, but really career changing for the, for the doctors as well. And really how the field is changing and what's going to be happening in the coming 10 to 15 years. So spinal muscle atrophy, really this grasp says it all. It is a genetic disorder. I'll discuss genetics in a second because it's autosome recessive. Generally, the parents have no idea that they, you know, their Children are at risk of it. The child is born completely normally, but within the first three months of life isn't the typical what we call S ma type one, but the typical type of sma, we'll discuss that in a second within three months of life. It's very clear that the child is not reaching any of the motor milestones. So they never are able to lift their head, for example, never get head control, let alone roll or sit or obviously walk. And again, the natural history of this disease is that by an average of what is it eight months of age, um the Children with this disease, the baby with the disease are either dead or on a ventilator. And clearly, when there is a disease, one doesn't put the baby on a ventilator. So, again, in this country, the child would pass away 80% of the time by the age of one year of age. Um And that's the ne of this disease. Let me talk to you about the genetics because it is really interesting and actually means we now have three different licensed and funded genetic therapies for this disorder. So let me talk through the genetics quickly. So the main gene that's missing in S MA is the S MN one gene. It's the survival motor neuron gene one. And as the name suggests, the protein, it produces the in one protein is important for the survival of your motor neurons. So not for the development, that's why the Children are born healthy, but for the survival crucial. And without that gene, the motor neurons die off very quickly and therefore you develop progressive weakness of all your muscles, which obviously isn't compatible with life. Now, as luck would have it, there is a backup gene called the S MN two gene. And it looks survival by neuron two gene looks very much like the S MN one gene. Um Except for the fact that there's a slight difference at instead of AC and exon seven, which means that when the RNA is being turned into M RN and then ultimately protein, 90% of the time exon seven is skipped. So you end up with exons 1 to 6 and exon eight versus all eight exons in the SMN one protein. About 10% of the time from the SMN two gene, you do get the exon seven included and that's a functional protein. And it's this 10% functional protein that allows, you know, that, you know, allows any kind of survival at all with 90% of it, you're missing exon seven and therefore have no which is not functional. Now, the interesting thing is as I looked at before, the most common kind of S is S MA type one. And genetically, these patients have the normal two copies of S MN two, of course, missing S MN one, which applies to all of mutations of both alleles, applies to all these patients. But with S MA one, the most severe form, you only have the normal two copies of S MN two. We do have patients with S MA type two. So they sit but never walk. Whereas S MA three are patients who walk but still have something before adulthood. It's a very rare type of S MA four with symptom onset is first in adulthood. And what they discovered rather is the more extra copies of S MN two. You have ie the more little bits of 10% functional protein you're producing the more mild SMA A you have. So patients with SMA four and again, I only have a couple of patients at Queen Square actually have eight or nine copies of SM two. Luckily. So they're producing, getting towards a reasonable amount of the protein. And of course, what that means is there's different genetic ways of curing this disease way. Number one, which came first is an antisense which we inject from the spinal cord, it's called spinraza and it basically blocks a little bit of a promoter here which causes us to skip. And so it basically promotes S MN two gene to produce the S MN one protein. And again, that's shown to be highly effective in S MA I'll show you our next slide. There's also now an oral agent wister plan, which we're also using in adults, which again is an oral agent that does the same thing. They've looked at different drugs and found one that helps promote the read through an inclusion, therefore, rather of exon seven. But maybe most excitingly, there's also a gene therapy which ignores S MN two completely and just provides the missing SM one gene. So it's an A ab nine vector S MN one is quite a small gene as you can see, only eight exons. So it fits into this vector. This vector is chosen the AAV nine because it loves spinal motor neurons. So basically it, you give it as an injection into the vein, it travels through the bloodstream to the spinal cord and then just sits there and of course, the other great thing about the spinal motor neurons or a bad thing for S MA, but a good thing for the treatment is they don't turn over. So once you've given one course of dose of treatment, there's little circuit of DNA, there just sits there on your motor neuron and uses the cells own machinery to start producing S MN one protein. And so we now have that as a single one of treatment injection in the vein. And this is what happens to the patients with that drug. So, rather than dying by 12 months of age, none of them die. And in fact, more than that, if you give it early enough, the patients will walk, they'll climb stairs, they'll essentially have normal motor milestones. And it is absolutely are fantastic. In fact, what we've discovered is if you wait till I have symptoms, you'll still get an amazing response, but not as good. So we're in the moment in the process of setting up a newborn screening program to genetically bind the patients who will have the disease and then treating them within the first few weeks of life. Of course, siblings, we can identify in advance and treat, but this will, most cases don't have siblings. As I say, most, most patients, the parents have no idea the carriers of this gene. Um And again, this will completely revolutionize the way we, we treat these patients. And again, these treatments, these gene treatments that rather than being the, what I consider the ambulance at the bottom of the cliff, you know, something that's gonna help predict your nerves a bit or make your muscles a bit stronger. Um, and generally don't work very well. That kind of treatment paradigm. We're looking at treatments that start right at the start and give you the missing gene or block the damaging gene or et cetera. And again, there are treatments now for hemophilia that work exactly the same way. But rather than the SMN one gene going to the spinal cord, it's the factor eight gene going to the liver. But you know, the actual treatment is the same. It's just you have a different truck if you like and a different cargo. Um And so really the possibilities of this are absolutely amazing. And neurology, again, we're at the forefront, but across all diseases, this is what's going to really change in the next few years and really revolutionize the treatment of these diseases. So that's perfect timing, I think. And what I wanted to cover again, I'm very happy to take any questions. But again, just briefly, I've talked about my journey to Queen Square. I've talked about the varied job that I currently have the exciting research that I do. But as I say, most excitingly, these gene therapies that are now coming into practice and certainly by the time your consultants still be, I would say 100s of these to be honest with you, the biggest challenge, of course, and again, you may have seen this xolex are referred to in the newspaper because you know, roughly speaking every month or two, if you read the newspapers carefully, you will see some reference to a mother and father who say my child just received the 2 million lb therapy. This zol that changes you from dying at 10 months to having a normal life. Roughly speaking, the list price for the taxpayer or for the government for the NHS rather is 2 million lbs. The treatments we're given for adults. The other ones, the risk of plan has been Raza a roughly speaking list price of 200,000 lbs per year. And again, yeah, so there are certain implications of these treatments but certainly it's extremely exciting time. I will stop there and I'm happy to take any questions about uh neurology, medicine, uh training in general training in the UK. I do um I do supervise trainees here. So I do know about the training system here even though I trained somewhere else. But again, very happy to take any questions, hopefully have some, maybe some some interesting thoughts. So thank you doctor m um anyone if you'd like to put any questions in the chart? Mm So doctor Ma question for me. Um I guess those who are interested in pursuing neurology, knowing how competitive field it is. What advice would you give them to strengthen their applications. Yeah, it's interesting. I kind of alluded to it when I was talking in that the particular time and place I was in New Zealand. It wasn't competitive because of the department. I just happened to be my home department actually. But certainly in London neurology is competitive. It's the same actually process now as for all the, all the specialties in that you need to get your train number. And again, there are a certain number of points assigned to different things you can do at different points in your career, you know. So writing a journal articles, first, the author gives you a certain number of points. What you'll discover if you look at the point system is not really very affair is the wrong word, but the points are comparable. So you to do a phd, you get, I don't know, three points or something, I don't know what it is. But again, you can look it up. Whereas for doing a presentation at a conference and a paper, you get three points or something, you can get the same points in different ways. So in regards to that, I think the most important thing is if you're interested in urology, number one, showing interest early because there's always opportunities to do extra things or go to conferences or write a case reports, do audits. And again, so showing that kind of interest and commitment is probably the most important thing. And yes, there are the points. But ultimately, you know, there's still a application process like any job. Um The second thing I would say, and again, I would say this genuinely is that, you know, although, you know, it was great to finish my training at Queens Square, you know, I think neurology is, again, neurologists everywhere are great. That's the first thing to say. I don't think there are really any bad neurologists in the UK. And so if you really want a gene, you at the moment again, I was talking to people replying at the moment, you have to weigh out which places you put first. But if you really want to do it and are happy to go somewhere smaller, then obviously, it's much easier to get into those places. Again, as I've said, there are huge advantages to going somewhere smaller. You come to Queen Square, you'll learn about all the random things, but not the most common things. So that's the other thing to say. There's a question that's related to that. Maybe I'll answer at the same time, which is doing a phd before becoming a REG. That's no longer, that's no longer the case that you have to. So we were kind of discussing this before we came on air, but 15 years ago, more or less to get a neurology number. Certainly in London you had to have a phd. Again, the system was different then it wasn't the point system. It was very much an application interview process. And that was just the way that it was. And as I say, the disadvantage of that, in fact is you end up doing a phd in something that you don't know you're interested in. And so one of my colleagues, for example, did a phd in motor neurone disease, but now doesn't do any motor neuron disease. And so again, this is a personal thing. I think research is extremely important, but you can combine research as you're doing your clinical training. And then there are also opportunities to do a one year research or do an MD or do a research fellowship for a year afterwards. As part of your cation, there are lots of opportunities for research because in some ways to be honest with you, I can say this and you don't tell anyone else. But a lot of the purpose for the p hds in the past was just to get people to do the research. It, you don't need to do a whole phd to be a good neurologist if you're going to be a clinical neurologist. So again, I would really think about where you think you want to be in 20 years time. And if research is going to be a major part of that and you want to work in a major London teaching hospital, then the phd at some point is going to be important. But even in the UK, there are more and more uh people, I work with registrars. I know now who have done pictures with us who have done at the end of their training. And as I say, there, there are good advantages to that. In fact, uh, are for different reasons. Thank you. We, um, I have quite a few questions which is always great to see. Um, I'll start with the first one. by Alice do gene therapies generally have better outcomes than drug therapies. So, II mean, again, it's, it's, it's, it's, it's, it's a, it's a, it's a term you use when you want to get funding in the sense that in some ways, gene therapies are a form of drug therapy, right? Um And you know, and diseases like Parkinson's disease where we don't know the cause we kind of can't give gene therapy. So we're stuck with in very common drug therapies. Um I'll give you a good example. So in, in, in muscle wasting diseases, there's a, there's a great drug um or great idea for a drug called Myostatin inhibitors. And myostatin is um AAA receptor or a, a AAA transmitter, uh uh which um um which blocks muscle growth. So it's basically a sign of the muscle you've grown enough. So if you block myostatin, the muscles grow and indeed, there's a, there's a, there's a breed of cow which has a, a genetic problem with the myostatin uh receptors and they end up with the most amazing muscles you've ever seen in a cow. Um And so the thought was, well, let's develop a therapy where, you know, we give myostatin inhibitors to patients with muscle wasting disease and their muscle get stronger. And again, if you try this treatment in healthy people, it works, their muscles get bigger. Um, but there's now been five or six different trials of my inhibitors in muscle wasting diseases and they've all been negative. And the problem is that, well, my opinion of the problem is that basically the body is an amazing system and it's already basically blocked that as much as it can to try and preserve its muscle because it knows it's going, you know, it's getting the signals that muscle wasting. So it's turning on all the signals to make the muscles bigger and turning off all the signals that make muscles smaller. So all those treatments have been negative despite being a drug that works in healthy people, if you see what I mean. So again, I do take this kind of ambulance at the bottom of the cliff approach versus stopping the problem with the source or for example, you know, a disease like my dystrophy. So there's a problem in the genetics where you have a train to repeat, but that causes problems in almost every organ system. So if you wanted to give a drug treatment to help the brain or to help the sleep or to help the heart, and we have all these treatments and we give them all, but in the end you're doing a partial job and you're doing it once the problem is already there. So, you know, I think gene therapies early are going to be a cure rather than just a treatment to slow things down. Ok. Um, thank you. And, uh, do you recommend a specialized foundation program to increase your chances? Um, you can do it through either route so you can do the academic program. I mean, they're even more difficult to get into. But again, this is, this is a personal thing. And again, obviously, I didn't, didn't come through the system, but looking at the other end, so again, I see people who become consultants and again, at the consultant level, you can choose some of my colleagues have done to get a say an MRC fellowship or NIH R fellowship where you're fully research funded and you do a little bit of clinical stuff on the side or you can take my route, which is to take an NHS job and build up your research time and do the research as part of that. The disadvantage of going down an academic route at any point is of course, if you don't get into the next phase, then all of a sudden you're without a job. And that's why regardless of what you take, I would encourage yourself to keep yourself as multiskilled or broadly skilled in your area as you can. Because if you end up as a, ah, as just an expert. And again, this is a Queen's square example because with the National service in a current paralysis, which is muscle channelopathy, and you've done that for your phd and then you've done a fellowship in that or academic fellowship. And at the end of that, you don't get to the next stage because every stage, there's people who don't get to the next stage, then you are going to have to do general neurology or other neuromuscular. And again, so you do need to keep those skills up throughout. Um But yeah, both routes are quite possible, but again, it's up to the individual. But I think the other thing to say definitely is don't worry or you don't feel like you have to go the academic route because you can go into the academic route at any point. And again, if you and again, really, again, this is a personal thing, but II didn't personally become a doctor to sit in a lab all day. So all my research was clinical. There are other people who became doctors that discovered, they didn't like that so much. They go into wet lab research and don't, some of them don't see patients at all actually. So I think as long as you are aware of what each option is and then take the best one for you, you can't really go wrong. I think that's a very good point. And um what are some ways of getting research as a medical student? Um So I have assisted. Uh don't, don't, don't contact me all of you. Um But basically, uh th think about the area that you're interested in. Um And again, when you're doing rotation, a teacher who, someone who's teaching, who's a really good teacher seem really inspiring and then just ask them and what you'll find about consultants generally, but particularly consultants who are active in research is we always have far more stuff that we've done or would like to publish than could be published. Um And so, you know, for example, you know, we have a whole lot of data that we've collected. And again, we've analyzed the basics of it and we've published that. But if someone said to me, they wanted to spend the year alongside their clinical work doing some research, then absolutely, they could do that and get a paper or two out at the end of it. If you have less time, then there's the opportunity to, as I say, do a case report or get involved in an audit or that kind of thing. Audits are really important for care. And again, you learn a lot from audits beyond just just what you set out to learn. So again, the best thing to do is to approach someone, I mean, to be honest, again, what often people are asked to write like myself, lots of journals will ask us to write review articles and we don't have the time to write them. So normally some are more junior. And again, there's no reason a medical student couldn't write the review article. And of course, it would be supervised and reviewed et cetera by the other people involved in the paper. But this is another great way to get, to get articles to get publications. Um but even just being involved in the research. So again, I would encourage and again, medical students have done this, you know, going along to the conference in the area that you're interested in, you know, whether that's neurology or whatever area of medicine you're interested in because that will really get you to understand how the research systems works and you can meet people, you know, um by their posters, I mean, the posters, these poster sessions, all these conferences where people stand beside the posters and if you walk around, you can talk to the more junior research people, obviously, the more senior people are often given the talks and you can chat with them over whatever, but you'd be surprised how often it's just that random kind of kind of link. You see what I mean? Like again, the consultant I worked with in Wellington had worked at Queen Square 20 years ago. And so I kind of knew all the people here and again, could write me a reference when I applied for the job. So really making those links is the most important thing. And as you just being interested, being interested and proactive, you'll be amazed what people will do for you because again, they'll think, well, this is someone who's going to deliver the worst thing for me would be to try and help someone for whatever the research project is. But at the end of it they get disinterested and don't finish it off. So, just showing their interest and et cetera is the most important thing. OK. And um one person has asked, um I think we talked about commitment to specialty for training um before um but they were asking if they, if you offer any observer ships um under yourself in mus neuromuscular diseases. And somebody else asked if, if the observer ships are present, if they are foreign doctors as well. Uh I think it dep it, it depends. So, yes, there is. Um we do have observers at, at, at, at Queen Square, but they're never um because of, I mean, again, we literally get emails every week. Um So they're always gonna be the ones who get observers. They're always gonna be someone who is not known to us directly but known to us in some way. So for example, we have, we now have all around the world. So we, we, we, we're part of the International Center of genomic medicine and Neo Disease, which has, has hospitals we're involved with in India, Turkey, Botswana, South Africa and Brazil. And somewhere else I forgotten um or, you know, different research projects were involved with people in a, you know, Australia, India, America, Canada, etcetera, etcetera. So most of the ones to be fair because of the number that we get that we accept. But because I collaborate, say, look, I've got this person who's really good. Can they come, come with you for a while? What I'd recommend doing if you're interested? Um again and, and again, and you is obviously getting to know the specialist where you are because they will have had links overseas or links to training or links to whatever. And that's going to be the most guaranteed way or not the most guaranteed, but the most high probability way of kind of getting that link and getting that in because what we really want again is not just for someone to come for a month because now there's unfortunately a lot of paperwork to do and a cost involved actually as well that the person has to meet, but for us to get something out of it if you're coming for three months or six months, and there's enough time actually to get you involved in a research project and to contribute as well. The other thing I would say again, because we do get these, get these ones where people just, well, it seems obvious to me that they've just written to 100 different people. And so if you are more doing like a cold calling type approach. Again, what I would suggest doing the ones that I do look at and reply to and try and get some advice, the people who are very specific, like I want to come to your hospital for three months because I'm really interested in the specific things you have there often. It's just like it's quite clear, they don't even know what most diseases are sometimes and say, you know, obviously attach a CVI, don't want to have to ask for a CV. And you say yes, I have GMC registration and therefore you can be more helpful. You just want to be an observer or um you know, that you need funding or because there are funding opportunities, you know, we we're short on doctors in the UK. So again, if you can get GMC registration, get an observer, you could come and work on the hospital and get paid for being here and you'd be surprised how many empty posts there are at Queen Square and for the right person who's interested and excited. And you know, obviously there's an interview interview process, but you can get a job at Queen Square, forget about an observer. But again, the difficulty then of course is giving your GMC registration um that you obviously need, need, need for that. But there's definitely these opportunities. OK, thank you. And um what's the would you know the criteria for applying postgraduate in New Zealand um in any field in general or also the criteria for doing internship in New Zealand. Uh it's quite straightforward really in the sense that in fact, the, the hospital I worked at in New Zealand at Hawke's Bay. Um, over half the medical registrars were, were British rather uh of, of, of, of, of some variety. It was a very cos, it was a nice sunny place and, you know, small but not too small. It was very popular for British doctors. And of course, because of the ratio, although a higher percentage of New Zealand doctors in training come to the UK. In fact, as a neurology trainer, you have to go overseas above your training because neurology isn't considered big enough these days, they let you go to Australia, but in the old days, it had to be somewhere in Europe or America. Um because we're a small country, obviously, we diffuse out. Whereas the other way around, a small percentage of doctors coming from the UK to New Zealand for a while would be a big proportion of the doctors in New Zealand. But yes, it's fairly straightforward. Again, you do. Now, it used to be that your registration was essentially automatic with the UK registration. And certainly that was the way in the other direction. I think that was still the case. Actually, I think again, if you've gotten through and got your registration and working as a registrar in the UK, you don't have to sit exams to work in New Zealand. But to be honest, you just slot into the job at what level you're at. And there's been plenty of people like myself in the other direction who have gone for a year and stayed for 15 years. Um So again, the two points of the real point of training in New Zealand, which is competitive is entering advanced training. So to do that, you have to pass your membership exams much like the UK for physicians. So you pass a written exam and then a vi a based exam, os based Viber based exam. And at that point, you apply for training in your. So as I said, less competitive than the UK. Incidentally, the training is considered equivalent but not automatically. So, so when I came to the UK, having trained most of the time overseas, I had to complete something called a certificate of equivalence of specialist registration, otherwise known as Ce Sr or AC A. And again, if you went through the training program in New Zealand, obviously, you wouldn't have to do that to stay in New Zealand, but you could come back to the UK and do it the other way around. But again, again, I can't speak from experience who obviously have gone the other direction, but there are 100s of doctors who do it and it's quite feasible to do for a year. I don't think the process is very difficult and certainly New Zealand, like the UK need lots of doctors. And again, I think it's a mutually beneficial process because, you know, again, it's, it's, it's, it's boarding and you're learning and wherever you end up as a doctor, you'll be a better doctor for it. And in the end, I think honestly as well, if you take a year out to do that and come back, it will be looked upon favorably in terms of your training, applying for training subsequently. I don't think it'll be looked upon unfavorably. You just have to time it. Right. The sorry, I missed this question above. But um one person says they heard you can get into neurology via I MT as well as A CCS. Is this correct? And if so do you have thought on one over the other? So I think that's what I was saying, you can go through the academic route or via the standard route and yeah, you can do it either way, one isn't considered better or worse. It just depends. I mean, to be honest, in my opinion, the biggest drawback of the academic route is it takes twice as long because you're only doing half time clinical. And again, you get a little bit of dispensation, but it does, you do see some very old people, not old but older people become a consultant at 35 or 40. So, um and again, you can do it either way. I mean, again, there's a question about when to do the phd as well. I kind of touched on that. I personally found doing a phd at the end. Very good. Other people do it earlier on. But I think actually it's quite difficult to do six months of research and then six months of clinical work because you never quite the clinical work and you never quite get proper into the research. So it depends on the project. If you have a very clinical project, obviously, you kind of can do them in parallel much better. Um But I personally think if you're interested in research, actually, your phd is a good way to go and then you don't really need to do the academic route. But again, both are good options. Thank you very much. Um I think we've answered all of the questions there. I'll just leave it for a couple of minutes. Does anyone have any further questions or anything that I didn't cover that you initially sent before? I think we've answered all of them, haven't we? I think he some um the same question around the phd. Yeah. So that's right. Thank you. Um Good. Thank you. OK. Uh Thank you very much, Doctor Mara. Um And thank you everyone for attending. Um It's incredibly useful and really great to get insight into his career. Hope it's been interesting. Yes. Yes. And so this session is recorded. So if you'd like to rewind it in bits or if you'd like to catch up then, uh feel free to do so and any problems. Um Please let us know. Thank you. Once again, Doctor Murray, thank you. Take care everyone. Have a nice evening.