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welcome everyone to today's mda engage pediatrics seminar thank you very much for joining us my name is nicole petrowski and i am the community education specialist here at mda i wanted to let everyone take a look at the agenda that we have today we have a pretty busy day as you can see we are going to start with dr catherine matthews here in just a little bit but i do have just a few housekeeping items before we begin our seminar so everyone is aware we are recording today's event and we will be posting it to the mda.org website for on-demand viewing in a few weeks and for those of you who are joining the event live we do have all phone lines muted we will be having a q a session at the end of each presentation so if you hover over either the top or the bottom of your screen you will see some webinar icons if you utilize the q a icon to type in your questions you can submit those whenever you have them you can submit them to host when that is necessary and then also you can feel free to use the chat feature you can also see that on your icons you can use that during this seminar if you have any comments just make sure you click on all panelists and attendees when you type in the comment if you just type on all panelists i will be able to be the only one that can see that and then you'll notice on the agenda there are a few small breaks between some speakers five ten minutes here and there so feel free you can just stay connected to the live broadcasts during those times but we will also have um that will also give you time to get something to eat or grab you know some water and that is also the time where i will be checking the next speaker's audio and video so you will hear me speaking so i am aware of that and then finally we will be sending out a brief survey afterwards and we would like to receive your feedback on what you heard today we want to make sure we are discussing discussing topics that are of interest and that are relevant to our community and so we use your feedback as a way to improve future educational events so thank you in advance for taking that five to ten minutes to complete that survey and also just so you know we are giving away two twenty dollar amazon e-gift cards for those of you who do submit um at the end of the seminar we're gonna be drawing two emails out of that group so that's a little enticement for you guys to do that now i would like to start off this seminar with just some information about mda and to give you a quick background on what it is we are and what we do we are committed to community education and believe in bringing our community together for opportunities to learn from specialists and have opportunities to connect with others this event is part of a larger mda engaged series with disease-specific symposia community education seminars such as this and also our engage community webinar series which are our long series we are committed to transforming the lives of people affected by muscular dystrophy als and related neuromuscular diseases we are able to honor this commitment by our work in two areas through innovation and care via our 150 plus care centers resource as a resources our education and recreational programs and then also our innovations in science with focus on research therapies and technology and we have been doing this for over 70 years we are an umbrella organization so we do support over 43 diseases in the neuromuscular disease space no other non-profit supports so many neuromuscular diseases and as an umbrella organization we have the benefit of leveraging key learnings from one disease to inform others and to learn more about the 43 plus diseases under our umbrella please visit nda.org and you could go under the about neuromuscular disease heading next the innovations and science section and this is where mda has made significant investments in advancing the treatments for neuromuscular disease we are the largest source of funding for neuromuscular disease research outside the federal government 1.4 billion since our inception mda's research is directly linked to approve life-changing therapies across multiple neuromuscular diseases we developed a first and only data hub to aggregate healthcare genetic and patient reported data and help accelerate future breakthroughs on this slide you can see the progress that has happened in drug discovery over the past five years in the neuromuscular space treatments are now available for periodic paralysis dmd sma als myacenia gravis and lens and we are proud to have provided funding to many of these treatments along the development process our mda mover data hub which stands for neuromuscular observational research is improving the ability of researchers and healthcare providers to enhance the care and management of individuals living with neuromuscular disease this is done by driving disease understanding accelerating therapeutic development and optimizing health outcomes mover collects clinical and genetic data from multiple neuromuscular diseases to improve health outcomes and accelerate drug development we are working this year to include patient reported data in mover currently seven mda diseases are in our database als sma duchenne becker fsh lim girdle and pompeii to expand this number we are going to be increasing the diseases that we cover in the coming years so that is something that we are actively working on and we are also actively working on getting mover implemented in as many care centers as possible mover only succeeds if we have the client population like those of you in the audience who take part if the mover registry is available at your care center and you have one of the seven covered diseases just mentioned please consider joining the registry and talk to your care center director now let's take a look at mda's innovations in care and we will start with our care center network we have the largest network of care centers for neuromuscular disease providing best-in-class comprehensive care and some of our speakers that we have today are those care center directors our care centers ensure multidisciplinary approach for patient care which provides patients the opportunity to see multiple clinicians in one visit and allowing for comprehensive coordinated care that help reduce the number of troops required to take to the doctor and you don't have to navigate your disease journey alone we're here to help our mda resource center is available to provide one-on-one support via phone or email for individuals and families looking for information about the disease covered under our umbrella our resource center is staffed by caring professionals and offer a unique perspective and support the mda community it provides information on the care center network some disease information community resources it also talks about our engaged series such as we're having today and then you can look at your screen we cover several other things as well our resource staff is available monday through friday from nine to five central time and typically are able to answer all your questions within one to two business days this is another great tool that's available at your fingertips and this comes up a lot in our in our talks you know where can we find a clinical trial so this tool provides a comprehensive list of currently enrolling clinical trials in the neuromuscular disease space the tool will walk you through some simple questions to direct you to the appropriate trials that meet your criteria when you share that and you can look at this tool on mda.org clinicaltrials we have a myriad of patient and caregiver resources which are available on mda.org and through contacting the resource center they include the quest magazine which is mailed to your home quarterly multiple print and online resources such as the carecovers guide teacher's guide and also disease specific information and you can sure to be connected to our mda blog which is called strongly if any of you guys like to read about blogs one thing mda is dedicated to is advocating for national policies and programs that accelerate the development of therapies and cures facilitating early diagnosis and treatment from day one and ensuring access to the covid19 vaccine and ensuring access to critical support and we also want to make sure that we are promoting independence together with advocates and families and volunteers and partners we ensure that the collective voice of our community is heard some of the current advocacy initiatives include accessible air travel newborn screening access to genetic testing patient-focused drug development meetings and increased federal funding for research we need you to join our advocacy network it helps the more families that we have involved thank you to those who who have already joined us as mba grassroots advocates and for those of you who have not please consider signing up today at mda.org advocacy or texting as you see on the screen 50457 to mda usa becoming a grassroots advocate means you will be informed on various ways you can join us in advocating for policies and initiatives that support the neuromuscular disease community you will receive advocacy alerts which lets you contact your representatives directly and share your stance on issues and you will be invited to take part in various advocacy issues and events sorry so we do have multiple ways to become involved with us from attending one of our educational events to share our social media posts to volunteering virtually and when it's safe to do so in person so the two areas to highlight on becoming involved with mda and join our organization by actively participating in one of our fundraising efforts so we encourage those of you who are interested in our mission whether you have a diagnosis that falls under our umbrella or are a parent a guardian or a loved one of someone who does please join us you will become part of a community of families researchers physicians and advocates who are all helping children and adults live longer and grow stronger you will also stay up to date on the latest information and education to help keep your family informed in the neuromuscular community you can visit mda.org join and sign up it'll take about five minutes for you to complete those steps you will ask yes a couple questions about you or your loved one's diagnosis and your contact information and we have multiple opportunities to become active in fundraising efforts our muscle log which many of you have participated in when we were able to do those in person it will be one day virtual or it will be one day live again but for right now our virtual event is on august 7th and throughout the day we will be celebrating with our national ambassadors hosting virtual pep rallies events and fellow participants we'll have breakout sessions for more information you can go to mga.org musclewalk and we are hosting of movement month in may where a focus on activity and fundraising will take place there will be chances to join live class with the mda community kickoff for that is may 1st and we will join each other virtually on may 22nd to celebrate what accomplishments were done that day and lastly with team momentum every mile you run walk cycle or move and every dollar you raise will help fund the important work we are doing for more information on team momentum visit mdateeam.org that's mdateam.org and so that is just a little bit of an overview of what we are about here at mda and if you aren't part of our organization i hope that um you definitely will consider joining us and we would look forward to that so thank you with that i would like to introduce our next speaker we are going to hear from dr catherine matthews who is a professor of pediatrics and neurology and the vice chair of clinical investigation for the department of pediatrics at the university of iowa carver college of medicine she completed all of her postgraduate graduate training at the university of iowa including pediatrics and child neurology residencies followed by a fellowship in medical genetics she was involved in the early efforts to map the gene for sshd and current research activities are focused on clinical aspects of muscular dystrophies with the goal of improving outcomes she directs the clinical project at the university of iowa's wellstone muscular dystrophy cooperative research center and is involved in multiple industry-sponsored clinical trials of potential therapies for neuromuscular diseases dr matthews runs an active clinical service and has been the director or the co-director of the university of iowa's mda clinic for over 20 years so thank you for being here dr matthews it is my pleasure to join you this saturday morning in the midst of a pandemic and record-breaking horrible weather so i hope everybody is warm and has water and food what i'm going to be talking about today is multidisciplinary care in pediatric neuromuscular disease and let's see i'm sorry i'm having trouble making my slides move there we go i'm sorry having technical problems this morning so first of all uh disclosures i have no pertinent disclosures um regarding today's talk although i do get funding from a number of different both organizations and industry sponsors so what i'm going to talk about today is what is multi-disciplinary care and why is it important what does multi-disciplinary care look like in the clinic i'm going to give you some potential models and give you our example and then because i knew i couldn't talk for 45 minutes on multi-disciplinary care nicole asked me to make a few brief comments on cobit 19 and pediatric neuromuscular disease since that is a topic that everybody is very interested in so i looked up definitions i mean i kind of knew what multidisciplinary care was but i looked up i hadn't really thought about defining it so i liked this definition multi-disciplinary care is when professionals from a range of disciplines work together to deliver comprehensive care that addresses as many of the patients needs as possible and i like a couple things about that it emphasizes the as possible it's professionals from a group of different um specialties and areas of expertise um to try to do the best thing that we can for the patient so why is this important for pediatric neuromuscular disease and i suspect that i'm preaching to the choir here i think most of you on the audience live with this and so you are familiar with why this is important but patients and families have enough on their plates you are navigating a lot of different things and anything that we can do to help minimize that need to navigate is beneficial and then optimal care of pediatric neuromuscular disease and adult neuromuscular disease is quite complex and no single person can do it or know it all so we really need to take advantage of the expertise of our colleagues just as an example a couple of examples here to emphasize the complexity of care this is just the um overview of the the sections identified in the duchenne muscular dystrophy care guideline that was published in 2018. so i'm not going to read them all because i've got several slides that look similar but um so these were the topics that had been um included in the 2010 guidelines and then they added five more topics in the 2018 guidelines so um you can see that you know no single person knows all of these different areas of specialty and then there are similar um a similar list is from the 2018 sma management guidelines very similar they've sliced and diced a little differently but again multidisciplinary care is required so from the mda website these are the potential members of an mda care center to try to meet all these needs of these complex diseases cardiologists nutritionists genetic counselors neurologists nurses orthopedists physiatrists physical therapists and occupational therapists primary care physicians are certainly an important partner although they're not usually sitting in our clinic psychiatry psychology pulmonologists respiratory therapists social workers and speech and language pathologists so um what i'm going to emphasize i think is that you will not see all these people every day every time you go to the clinic but these are all people who may be involved in a child's care o how does this all fit together so i liked this quote from the sma guidelines and it is written about sma but applies to all of the diseases that we care for so it says sma is a complex disorder involving different aspects of care in the past families had to coordinate all the assessments and visits but it's now recommended that this should be coordinated by one of the physicians typically a neurologist or pediatric neurologist in sma who's aware of the disease course and potential issues so the way i put this together is the in general the primary neuromuscular doctor has the greatest knowledge of the breadth of the diseases so they um are the one who knows the the range of complications the kinds of people who should be probably involved and then they work together with the patient and family to assure that the appropriate other specialties are involved in care it each stays at the stage of the disease and the neuromuscular physician recognizes and defers to the expertise of each team member because as i said we cannot know everything so i'm going to spend a little bit of time on the fact that the mda clinic one size does not fit all and your experience in your mda clinic may be different than what you see online from somebody else's and there are lots and lots of reasons for this every institution state and region is unique patient populations vary there are what i call boutique clinics where most of their patients fly in from other areas of the country that's a very different setting than a clinic where many of the patients have more limited resources and the the hopefully the provision of care will be this as good in both of those but the clinics are going to look different another example is rural versus urban the um you know if everybody lives within a mass transit of the hospital you may be able to provide all of your care in one setting whereas if people live six or seven hours away you have to be a little bit more creative about how you operate your program and i'll talk a little bit more about that later state and institutional resources vary so similarly to where i talked about boutique clinics versus clinics that have less resources going to a state university clinic is going to be potentially different than going to one that has extensive private funding um you have to multi-disciplinary clinics are expensive so you know if you have a bunch of specialists and i by specialists i mean i'm including physical therapists and social workers a bunch of healthcare professionals who are um sitting in a clinic and may not always be seeing patients at the same time that is less efficient than if they were in their own clinic and they were scheduled every half an hour to see a patient so there are there are expenses involved in multidisciplinary clinics that make them more complicated to run the mda support while it no longer goes directly to patients is helpful in offsetting some of the costs of having for example a social worker available in the clinic or having a physical therapist sitting in the clinic where they may not see a patient every 30 minutes or every hour like they would in their own their own practice so the resources vary the mda support helps offset some of those resource demands but um but that's another reason that your clinic may look a little different than somebody else's clinic um i guess another way expense is that is huge is something like a cardiologist cardiologists are very expensive doctors and um so sometimes they will sit in a clinic and see you know five of the 10 patients scheduled that day but sometimes they will not be able to be able to just be sitting in your clinic um and then another way that um clinics differ is that the personnel roles may differ so the the the best example i think is the who runs who serves as a clinic coordinator so in some place cases it may be a nurse um a nursing assistant or a licensed practical nurse in some cases it could be a staff nurse in some cases it could be a nurse practitioner in some cases it could be the a genetic counselor in some cases a physical therapist may act as a clinic coordinator so each institution is going to have people with different skills skill sets and capacity to do any given role so again your clinic may not look like the next one or if you move the clinic you move to may not look exactly the same as the one you attend now turnover illness responsibilities that other clinic that clinic personnel have can temporarily change your experience we have our our nutritionist is out on maternity leave so while we have a substitute nutritionist she doesn't have the same level of expertise in neuromuscular patients that our usual one does similarly unfortunately people do not stay in the same job their whole lives so when we have turnover there's often a learning curve while we bring another specialist sort of up to speed on this particular specialized patient population so sometimes you have to be a little bit um patient as we deal with the demands of um just living in the real world which we're all um really quite used to at this point i think um another thing that has changed that i've certainly seen over the many years that i've been doing this is that clinical care changes and so the makeup of what is needed in the neuromuscular team might change the expensive new treatments that we are bringing in are fabulous but they also mean that we need different levels of both pharmacy and administrative support for example as one example there and then finally the the patient level needs are certainly highly variable different diseases need different team members and that's um so for example not all muscular dystrophies involve the heart i've talked about having the cardiologist sit there um but some muscular dystrophies don't affect the heart they don't need a cardiologist and certainly when we talk about all the different kinds of diseases that can be seen in one mda clinic you have to tailor the team members for the individual disease there are obviously different needs at different stages of the disease the four-year-old with duchenne doesn't have the same needs as a 14 year old with duchenne and then the prime if you have a primarily adult neuromuscular clinic those needs are not necessarily the same as a pediatric clinic voc rehab may be more important in the adult clinic whereas managing the school requirements are more important in the pediatric clinic there are a lot of different models for multidisciplinary care the goal again of of all of them is to minimize the burden on patients and families while optimizing care um i these are models off the top of my head i'm sure that there are others that um that i didn't summarize here but and this is sort of like how does the day work that's what i mean by model so one is that the patient comes to the clinic they're put in one room and then various clinic members go in and out and they're relieved maybe to go to the bathroom but basically they stay in one room and we move in and out in some settings that doesn't that's not possible and the patient needs to travel from space to space in a single day ideally within a building or nearby buildings um there are some situations where they a provider is not available on the day as the mda clinic and so appointments may be facilitated or helped to be coordinated by the clinic coordinator but they occur on different dates or maybe in a different building a different facility altogether some families prefer to get some of their care locally and that then can be coordinated with the primary neuromuscular clinic so for um there are sometimes that that families there are a lot of reasons that they may want to get some of their care locally and then i think probably the most common is the hybrid of any or all of the above so we where we try to personalize the delivery of multi-disciplinary care within the limits of what is available for the institution and clinic and what the family needs where they live how this is going to work out best for them i think most of us prefer to have a lot of the care done at one institution so that we have ready access to all the medical records so that we can talk to our colleagues um about specific aspects of care it's a little bit harder when you don't know the pa patient the person delivering the care if it's done locally um but that is not always possible whoops back here we go so um another aspect of multidisciplinary care is the balance of optimizing care with the potential for exhaustion or information overload so the information overload i'm i always worry about that when particularly very early in the disease when we are um trying to you know you're having somebody who just learned a disease and they're meeting with the neurologist and the geneticist and the physical therapist and the social worker and they are overwhelmed with data so trying to make sure that we don't give too much information i think people quit hearing anything after about an hour um on the other hand things have become a little bit different because most patients even coming in with a new diagnosis have done a lot of reading online so they actually come in with more questions but it's still a balancing act and then there's patient exhaustion patient and family exhaustion so um just when i read through the 2018 duchenne care guidelines i thought that's all wonderful but also very intensive so i put together an estimate which i considered a fairly conservative estimate of the amount of time that each of the things that would be listed for a 14 year old with duchenne who took a mental health questionnaire screamed positive on that and so needed the follow-up and the estimated time for their clinic visit was 400 minutes if we added in some lunch that would be 460 if we gave them 60 minutes for lunch which was 7.7 hours which is a really long time to be in clinic to be going through testing to be hearing new hearing information um especially if you have to drive there and drive back so it makes a very long day so again depending on the family the stage of disease we may break those up into more than one day um and again try to make sure that that we are scheduling with the family's preferences in mind oops so just as an example i was going to talk a little bit about how we do our clinic um we are always in evolution and i will say that um you know there we are i give this as an example because it's the one i know not because it is ideal in every way there are always things that i wish we could improve on just by way of introduction for anybody who is not familiar with iowa we're right there um the population is about 3 million the population of pigs is 20 million for reference so if we were taking care of pigs we would have a different problem um it's about six hours to drop to drive from border to border so our families are driving for five to six hours to come to iowa city which is over about there um our clinic sees all ages so the way we do transitions is that we follow patients into adulthood but we transition their cardiology pulmonary endocrine and other subspecialty care so um that way if they if somebody does end up needing to go into the hospital or something they have adult providers who are familiar with the disease and are able to take care of them and we do include both diagnostic assessment and long-term follow-up in the clinic i'll talk a little bit about outreach clinics too oops every single time so in our clinic these are the people who are available to see patients on the same day and typically see almost every patient so the nurse or genetic counselor christina trout our physical therapist shelly our nutritionist when she's not on maternity leave a social worker a respiratory therapist a genetic counselor is available and then christina is also trained in genetic counseling and i also have training in genetics so we have a genetic counselor available she can come down to clinic she is not necessarily sitting there to see every patient we've added a pharmacist who is available in every clinic she again doesn't see every patient patient certainly but is very helpful with um helping to manage and answer questions about complex and very expensive drugs that have become available for our patients and then myself so those are the people who are sitting in the neuromuscular clinic oops every time and then um in the same space we have pediatric cardiology pulmonary and endocrine so in general in our setting the patient sits in their room and sees all of the people on the previous slide they need to see cardiology pulmonary and endocrine often in the same room occasionally they'll be moved to a different room in the same clinic but often in the same room so um again those can be very long days and then orthopedics in our settings so this is just i bring this up because it's an example of how um despite our efforts to make everything as seamless as possible for our patients in our case um i do clinic on tuesday the orthopedist who specializes in scoliosis surgery operates on tuesday and he's had his tuesday operating room day even longer than i've had my tuesday clinic and that is not going to change so patients who need to see orthopedics or follow-up of their scoliosis either need to stay overnight and see them a different day or come back on a different day and then um psychologists we have both a healthcare healthcare psychologist who can help with healthcare adjustment and and stress management and then a psychologist who has a particular interest in the neuropsychological aspects of some of our disorders and does neuropsychological testing those are also not available on the same day as clinic um in general and this is one of those situations where um working with a local provider is often helpful if somebody does need counseling that's usually done once a week that can be in a state like iowa which is rural and we are not central it's really better done probably with a local provider there are a lot of other key personnel so we have a staff nurse who doesn't sit in clinic but helps with doing phone management and all helps prior approval and all those other things that happen outside of clinic the schedulers are critical we are often trying to schedule six or seven appointments coordinated in the same day they are a huge help we have an administrative assistant whose primary goal is to help manage our complex drug programs and then we have the research team shown here with coveted cove protection and isolate and uh social distancing who are available to talk to the family in clinic or available after clinic by phone so i mentioned outreach clinics because we are a rural state this is where iowa city is um we offer outreach clinics where we drive to these places that have stars so from one to two hours away to try to minimize the burden on families um wanting our care now outreach clinics we can't take everybody so this is who goes with us to outreach clinics we have a neuromuscular physician a nurse coordinator physical therapist and often a student and or a resident um these are very busy days um so i will typically leave at six and get home around eight at night on these days sometimes the outreach center will have some additional ancillary help that is there but often we'll work either with local people do phone follow-up with other team members or we'll help to arrange visits with local providers so it's another example of the balance between trying to provide patient convenience and the one-stop shopping again we don't have the full team with us when we go to outreach clinic and finally i wanted to just mention telehealth i think everybody knows that there's been a rapid growth in telehealth thanks to covet 19 and for a period of time we were all able to there was um reciprocity between states so we were actually able to for example i was able to provide telehealth to a patient in illinois for example um that has restricted back so that now you can only provide telehealth for a patient who is physically in the state that you are um that you are licensed for um we have done such peculiar things as having patients drive across the border and do telehealth by phone but um it is ideal for those who have limited ability to travel or during severe weather li e we've had this year um there are drawbacks um you know it's you can't do an exam it's really hard to do some of the physical therapy things that are so important i even though um [Music] the video we've gotten much better at video i think that it's not the same as being able to see somebody in person there obviously you can't do echocardiograms and pulmonary function testing so i don't think telehealth is going to replace in-person visits in neuromuscular disease but it is helpful in specific situations um so how exactly a multi-disciplinary telehealth visit works i think that is still an evolution i know a number of colleagues who will schedule you know you meet with your physician and your physical therapist then your um social worker then your clinical coordinator some variation on that theme um um [Music] other times you may um meet with your physician and then do phone follow-up on a separate day or as needed with some of the other um services in the clinic and then i'm sure there are going to be many other approaches to this over time so this is really i think still in evolution so to sort of summarize this part um what can a patient expect from a multi-disciplinary neuromuscular clinic you can expect assistance in getting your own or your child's medical needs met despite complexity and i wanted to emphasize again that this does not mean you will always get what you want we all do the best we can but it's it's sometimes just not possible to get the appointment at the time you want when you want it you should have a contact person who can help you work the system when i have family members who are ill i am blown away that anybody is able to work in our health care system without having a medical professional in the family so for anybody who doesn't have a medical professional in the family you should have somebody a name and an email and a phone number or you know my chart a way to get in touch with somebody who can help you work your way through the system when you have a chronic disease you should have a health care team who is familiar with the needs of neuromuscular diseases and again every cardiologist is not going to know the details of your particular rare form of muscular dystrophy um the the specialists may or may not know the details of your disease but they should be familiar with the needs of people with neuromuscular disease they should the cardiologist should not be asking the person in the wheelchair to do a six-minute walk test um they should recognize that although that may be helpful in some of their patients it doesn't apply to this one and then you should get assistance with navigating and by finding the resources that are available to help defray the costs and the additional stresses of living with neuromuscular disease so um and here i'm basically talking about a social worker or somebody who can fill that role there are a lot of services out there there's a lot of paperwork that you have to fill out i am totally ignorant of it and i am deeply grateful that i have social workers and nurses who understand that i don't see how families do it so you should be able to have somebody who can help you and then you should have confidence that your team is knowledgeable and is doing their best to help you so um this is a picture that christina trout put together of our neuromuscular team congratulating our covet era graduates wearing our face shields we are socially distanced outside at the time so we don't have on masks but um we work with a great team so um very briefly here just a couple of things that come up in clinic with uh with regard to covid19 and our neuromuscular patients in general my neuromuscular patients are used to social social distancing distancing they have been doing this for flu season every year for a very long time reminders from the cdc about how you minimize your risk the pediatric age group is in a relatively lower risk now um so this is from the cdc website yesterday i think um and shows that the number of cases of covid19 this is just cases that have tested positive is really quite a bit lower in the zero to 18 age group than it is in the other age groups and then most importantly is the risk of severe copa 19 is lower in children than it is in adults so although people with neuromuscular disease may have respiratory or cardiac complications the probability that a child is going to get severe covered 19 is way lower and it's this bracket way way way lower than older people so we are fortunate that the pediatric patients are at lower risk for severe disease anecdotally the patients that i have had even who have been quite weak who have tested positive for coping 19 have done quite well they have not knock on wood ended up in the hospital and my colleagues around the country largely support similar experience so i think that while this is always this has been an incredibly stressful time for parents of children with neuromuscular disease there is good news um but it clearly doesn't mean we can relax and maybe part of the reason that they have done well is that these families have been very proactive and very careful um i had one family point out to me that the power wheelchair acts sort of as a six-foot barrier and social distancing um approach um so coveted 19 and vaccination i think everybody knows this but the pfizer drug is approved for those 16 and over the moderna drug is approved for those 18 and over um clinical trials are in progress and i just highlight here when we might near hear something and this is from the new york times source of all medical information um so the pfizer is hoping to get results out first quarter of this year i have not heard that anything from them yet but this will be for children ages 12 to 15 and then following this trial they'll lower it down to younger children so assuming that it's safe and effective so um stay tuned first quarter we should be hearing something by the end of march moderna is still recruiting for similar ages although a little bit higher um and data is the point is expected sometime this summer so um trials are in progress in children and i think that hopefully by this time next year we will be vaccinating children caretakers of children with significant health care needs can get vaccinated and so parent that includes parents so if your child does is in a high-risk category i you have to look at your local requirements but in many states parents are able to get vaccinated covert 19 in school a balancing act children need to be with their peers they need that normal development they need to learn social skills mental health we are reading a lot about teenagers and how devastating this has been for mental health on the other hand some of my children say this is great because i prefer to be at home so you have to think about what's best for your child another issue is that most children make better academic and better academic progress in person on the other hand the risk of getting covet 19 is higher if you attend school than if you're living in a covered bubble so everybody has to think what are the rules in your school is are masks mandated or is it free for all is there do they attempt social distancing do they do any tracking of disease what's the level of virus in your community and then how's your kid doing how are they doing in school how are they doing emotionally so take home points follow the cdc guidelines balance your child's mental and academic health with the risks in the community and most importantly if you're offered a vaccine say yes sometimes people are offered one and say no because my aunt tilly probably needs it more than i do your aunt tilly's not gonna get it if you say no so if you're offered say yes so with that i will thank you for your attention thank you to the team the core neuromuscular team that i work with who are wonderful are my various funding agencies and i think we have time for some questions thank you excuse me thank you dr matthews we did get a couple questions that have come in um what is the role of the endocrinologist that's someone that this family has not seen okay so the endocrinologist often at least in my practice comes in relatively later in disease course they manage um osteoporosis treatments so if all all neuromuscular patients who have significant weakness have increased risk of osteoporosis and then for children who are on things like steroids that risk goes up so that's one major role of the endocrinologist another thing that they will address is for older boys who are on chronic steroids they can have significantly delayed puberty and may benefit from testosterone's supplementation so that is another thing that the endocrinologist will address those are probably the two main uh roles osteoporosis management and puberty okay and another person has asked how often does a patient need to attend a clinic loaded question yeah there's an interesting question so um i think some of it depends a little bit on on the it depends on the disease it depends on the disease state it depends on um the individual person so there are some diseases that are extremely slowly progressive and early on have very minimal needs and i may check in with those patients once a year occasionally once even once every two years or 18 months many patients have diseases that questions come up issues come up things are changing and we see so every six months is a very common time time frame um but there are times particularly maybe early and after diagnosis when families just have a lot of questions and i may see people every three months for a period of time or if somebody's going through a period of rapid change and we just can't do everything in one day so they're like so many issues there is not one size fits all so you have to tailor it to your situation um okay and then when a family does see a multi-disciplinary clinic are they to assume that all doctors are having a discussion about their appointment at the end of that day that is an ideal world okay um i would love to tell you yes on the other hand um that is not always the case we do i think we mostly try to if there are big issues we'll try to coordinate that but no most of us don't have the opportunity to sit down at the end of the day and talk about the patients some clinics i think do we find it hard okay um we have a question that has come in and i'm not sure if you're going to know the answer to this do you know if any duchenne clinics in the midwest have fibrogen for heart and lung available the fibrogen clinical trial i believe so yeah so um i can only answer for myself we are in the process of opening okay but i don't i don't know what the other sites are i would advise looking at either the mda clinical trial finder tool or clinicaltrials.gov okay all right and coming up next we are going to hear from dr parker go she's going to talk about research and clinical trials so we will also delve into his talk as well so thank you very much dr matthews for your presentation thank you