Duchenne Muscular Dystrophy: Diagnosis, Support, and Hope – PediaCast 604

Show Notes

Description

Dr. Megan Waldrop visits the studio as we consider Duchenne Muscular Dystrophy. This genetic condition remains a serious diagnosis, but there is also real progress happening in how we diagnose, treat, and support those living with this condition. Tune in for details!

Topic

Duchenne Muscular Dystrophy

Guest

Dr Megan Waldrop
Pediatric Neurology
Nationwide Children’s Hospital 

Links

Pediatric Neurology at Nationwide Children’s Hospital
Muscular Dystrophy Clinic at Nationwide Children’s
Muscular Dystrophy Association (MDA)
Parent Project Muscular Dystrophy

 

Episode Transcript

[Dr Mike Patrick]
This episode of PediaCast is brought to you by Pediatric Neurology at Nationwide Children's Hospital.

[MUSIC]

[Dr Mike Patrick]
Hello, everyone, and welcome to another episode of PediaCast. We are a pediatric podcast for moms and dads.

This is Dr. Mike coming to you from the campus of Nationwide Children's Hospital. We're in Columbus, Ohio. It's episode 604.

We're calling this one Duchenne muscular dystrophy diagnosis, support, and hope. I want to welcome all of you to the program. We are so happy to have you with us.

You know, Duchenne muscular dystrophy is a genetic condition that affects muscle strength and development, most commonly in boys. It's a diagnosis that has received a lot of awareness and attention over the years, especially around Labor Day weekend, when Jerry Lewis and the Muscular Dystrophy Association held their annual telethon. While Duchenne muscular dystrophy remains a serious condition, there is also real progress happening in how we diagnose, treat, and support children and families impacted by this disorder.

Today, we're going to explore what parents need to know from early signs and diagnosis to treatment options, daily life, and the continued promise of research and breakthroughs. Of course, in our usual PediaCast fashion, we have a terrific guest joining us in the studio to discuss the topic. Dr. Megan Waldrop is here. She is a pediatric neurologist at Nationwide Children's. Before we get to her, I do want to remind you the information presented in every episode of our podcast is for general educational purposes only. We do not diagnose medical conditions or formulate treatment plans for specific individuals.

If you're concerned about your child's health, be sure to call your health care provider. Also, your use of this audio program is subject to the PediaCast Terms of Use Agreement, which you can find at pediacast.org. So, let's take a quick break.

We'll get Dr. Megan Waldrop settled into the studio, and then we will be back to talk about Duchenne muscular dystrophy. It's coming up right after this. 

[MUSIC]

[Dr Mike Patrick]
Dr. Megan Waldrop is a pediatric neurologist at Nationwide Children's Hospital and an assistant professor of pediatrics at the Ohio State University College of Medicine. She has a passion for supporting parents, patients, and families impacted by Duchenne muscular dystrophy. That is what she is here to talk about, the cause, symptoms, diagnosis, and the evolving management of Duchenne muscular dystrophy. Before we dive in, let's offer a warm PediaCast welcome to our guest, Dr. Megan Waldrop. Thank you so much for stopping by the studio today.

[Dr Megan Waldrop]
Thank you, Dr. Mike. It's great to be here today.

[Dr Mike Patrick]
Yeah, we are really happy to talk to you. And I think this is one of those rare diseases that most people have heard of. And I think a lot of that is because of the telethon that Jerry Lewis did back in the day on Labor Day weekend.

And so, it really got a lot of awareness that other rare diseases may not have enjoyed in the past. But just so that we're all on the same level playing field starting out, what exactly is Duchenne muscular dystrophy and what causes it?

[Dr Megan Waldrop]
Yeah, so Duchenne muscular dystrophy is actually the most common muscular dystrophy in children. And it is caused by a gene mutation. It's a mutation in the DMD gene.

And it results in unfortunately, destruction and degradation of the muscle over time. And it affects the muscles that are responsible for walking and doing everyday activities and affects the muscles eventually that are responsible for breathing. It can also affect the heart muscles.

It can affect the muscles around the stomach and the bladder. And then sometimes it also can affect the brain. There is actually some effect in some boys and the cognitive function and mood and behavior.

So, it's a very significant condition. And sadly, it does progress over time. And so, it is life shortening.

Yeah.

[Dr Mike Patrick]
Now, when you mentioned that it is genetic, we have talked on this program before that genes code for proteins. Is there a particular protein that is an issue with Duchenne muscular dystrophy?

[Dr Megan Waldrop]
Yes, the gene is responsible for allowing the body to make the dystrophin protein. And the dystrophin protein is actually present in muscle cells. And it is responsible for the structural integrity of the muscles, meaning our muscle cells when we run and jump and play or weightlift or do other activities tend to break apart and then grow back and get bigger.

And so, the dystrophin protein is really important for how that happens. And so, in boys with Duchenne muscular dystrophy, unfortunately, that process can't be done quite properly. And so, when the muscles do break apart, it's much harder for them to come back together and build and grow.

And so instead, they kind of decline over time.

[Dr Mike Patrick]
And you mentioned boys a couple of times here. Why is it that boys are more affected with this? And just how common is it?

[Dr Megan Waldrop]
So, we think the incidence, it affects about one in 5,000 boys. And we think about 20,000 babies are born worldwide with this. And the reason that I say boys is because the gene, the DMD gene is on the X chromosome.

And it's considered an X-linked condition. So, in boys, they only have one X chromosome. So, if they have a mutation or a problem with that gene, they will be affected.

Interestingly, on occasion, girls can be affected, but it's very, very rare. And it's due to some unique genetic mechanisms that I don't know that we need to get into today. But I think it is important for any family who has a family member with Duchenne that is a boy to be thoughtful and perhaps ask their doctors about if any of the women or girls in the family should be evaluated.

[Dr Mike Patrick]
Gotcha. So, when we talk about an X-linked disorder, girls have two Xs. So, you would have to have, in simple genetics, and I know that these days genetics is not nearly as simple as we once sort of envisioned that it was.

But when we think about recessive versus dominant, this must be a recessive one. If you have one good X, if a girl does, and I say good, meaning that does not transmit this disease. Is that why, then, that girls are sort of protected because it's more likely that they'll have one X that is not affected?

[Dr Megan Waldrop]
Yes. So, they typically have a good X or whatever you want to call it, a typically functioning X. And so, there's weird things that can happen where the good X can have problems, too, or the good X can be hidden.

But yes, it's kind of thought of to be like a recessive condition. And since the boys only have one X and there's a problem on that one X, that's why they have it.

[Dr Mike Patrick]
Now, what are some of the early signs of Duchenne muscular dystrophy? Like, what would sort of raise a red flag for a parent or a primary care doctor that something's up?

[Dr Megan Waldrop]
Yeah, so it's not immediately noticeable, even though the blood work or the blood may show some signs of it. When you're just looking at the child after the child's been born, they will look pretty typical. No one will really notice.

As the child continues to grow, that's when you might start to notice some things. For example, they might be a little bit delayed in their sitting, though not most of the time. Typically, what we see is that they might crawl differently.

So rather than crawling on all fours, they do something that looks like an army crawl. So, they kind of drag their legs behind them a little bit. And again, this is not all of the time, some of the time.

They often have a little bit of a delay in walking. So, you know, and it might be just on the later end. You know, it's not always totally clear.

They also might get up off the floor a little bit differently. So, once they start getting to the point in their development when they can start climbing and standing up, it might look different than other children. It might look harder.

And then also, oftentimes, the calf muscles look really large. And then eventually when they get to running and climbing stairs, across the board, you'll notice that climbing stairs looks different and running looks different in these boys.

[Dr Mike Patrick]
When I was in medical school a few decades ago, there was a thing called the Gower sign where when they're in a sitting position and they're low on the floor and then they go to get up, they kind of walk up their legs with their arms, sort of helping to support their weight to get in an upright position. Is that something that you'd mentioned that they may get up a little bit differently? Is that what you were talking about?

And is that something that you see very often?

[Dr Megan Waldrop]
Yes, that is what I was referring to. So, they need, they have weakness in their hip and butt area. And that's really what helps the boys get up or anyone get up off the ground pretty quickly.

And so, because of the weakness there, they need to use their arms or other parts of their body to help them get up. So, their legs might be really far apart. They might use their hands to push up off the floor and then push up off their legs.

They might always go to a piece of furniture and use their arms to help them get up that way.

[Dr Mike Patrick]
So, the muscles and muscle weakness and distinctive pattern is going to be the main thing that we see. But you also mentioned that it can affect the brain as well. First, is that because of that dystrophin protein or is there some other mechanism happening that is going along with it in those kids?

[Dr Megan Waldrop]
It really is due to where the dystrophin protein is. And so, it's also present in the brain. And so, for certain mutations, they can cause troubles with the dystrophin that is also present in the brain.

It's not every single mutation, but it is some of them. And so, we like to ensure when we meet these families that we let them know they need to have an evaluation to assess for any cognitive or emotional issues as well. Because dystrophin in the brain is very important for learning and emotional regulation and other important kind of everyday cognitive functions.

[Dr Mike Patrick]
And so, what sort of learning differences or behavior challenges might we see in increased numbers in boys affected by Duchenne muscular dystrophy?

[Dr Megan Waldrop]
Approximately a third of boys with Duchenne might have a lower IQ score. So, they might have some learning difficulties. This can be difficulty reading.

This can be difficulty with writing. This can be mixing up letters, difficulty with calculations. And so, it's really important to have an assessment either at the school or through a neuropsychologist.

And that's something that we do pretty regularly in our clinic now before they enter school so we can make sure when they get to the school age and they're in school; they have any assistance that they need to really thrive. In terms of emotional and behavioral, there is an association with autism spectrum. And so, some of these boys do have a range of spectrum of autistic features.

There's also very high incidence of anxiety and depression. Also, sometimes ADHD and even things like obsessive compulsive disorder. And then just because this condition is so severe and eventually they start to look so different from their peers, I don't think it's unexpected for anyone that they can have trouble with peer interactions and become a little more anxious and nervous about social interactions and may tend to want to stay home more versus get out into the world.

And so, we really encourage trying to find peers who look like them either through like MDA camp or we do have like, what's the word I'm looking for? Peer-to-peer kind of linking up that we can do through Nationwide Children's to help them find individuals who look more like them.

[Dr Mike Patrick]
Yeah, really support group basically and other folks who are going through the same things that they're going through is always helpful. So, if we suspect this based on these clinical signs, then how is the diagnosis actually made?

[Dr Megan Waldrop]
We would send either blood or saliva for genetic testing. And the genetic testing is actually very, very complicated because the DMD gene is quite large and there are several, several, several different mutations that can happen to cause this. And so, we have a tiered approach to genetic testing and usually we get an answer with the genetic testing.

Sometimes it's such a rare or such an unusual or unique mutation that we actually need to do either a skin biopsy or a muscle biopsy to really help us determine if this truly is the cause.

[Dr Mike Patrick]
Is there a way to diagnose this before symptoms begin? You know, there are a lot of conditions that are checked for with like a newborn screening. So, you kind of get an idea that the problem is there before maybe symptoms occur.

Is that true with Duchenne as well?

[Dr Megan Waldrop]
Yes, it is. Even before newborn screening, which we just got for Duchenne recently, a lot of this tends to run in families. And so, we would have a lot of mothers who knew they were carriers.

And so, when they got pregnant, they would have testing done to know. But now in Ohio, actually for about two years now, I think April 22nd, 2024, is when we started newborn screening for Duchenne. But there's a caveat to that because it's not genetic testing for Duchenne.

The newborn screen is just checking a muscle enzyme level, which is called creatine kinase or CK, to see if it's higher than it needs to be or should be. And because of that, it is just a generic type of screen. So really, it's newborn screen for muscular dystrophy, but we're really using it to kind of highlight the need for earlier diagnosis in Duchenne.

But we are also diagnosing some other conditions since this started.

[Dr Mike Patrick]
So, it's really a marker that, hey, we may need to do genetic testing to try to figure out what's going on. But it doesn't mean that if that's elevated, you have Duchenne muscular dystrophy, you may need to get the genetic testing then after that screening test is positive.

[Dr Megan Waldrop]
Correct. When the screen is flagged as risk for Duchenne, they are then seen in our clinic. They have a thorough assessment, both by a neuromuscular provider and our physical therapists.

And then we decide what testing needs to be done. And most of the time, it ends up in a diagnosis of Duchenne, but we have had a few other different diagnoses as well.

[Dr Mike Patrick]
And as more states move forward with this, and here in Ohio as we do it, I think one thing that's going to be important for parents is that that CK enzyme being elevated, it's not 100% in terms of catching every single case. And so, you can't go back and say, well, the newborn screen was normal, so it cannot be Duchenne muscular dystrophy. But that's not necessarily true, right?

We still have to look for those clinical signs and think about it and test the kids that were worried that could be affected, even if their newborn test was normal. Is that correct?

[Dr Megan Waldrop]
That is correct for the reasons you state. And then also that the tiered approach. So, it's very complicated.

And we have babies who are born at different ages, some babies who are born very early. We also have babies that are born at different weights. And we have different cutoffs for each tiny little category.

And we're not entirely sure that the cutoffs that we've chosen are perfect. And so, we know that we're probably going to miss a couple. And so even if the newborn screen did not identify high-risk for Duchenne, if there are any signs of crawling differently, trouble getting up and down stairs, trouble getting up off the floor, the child should be evaluated.

[Dr Mike Patrick]
And from a primary care perspective, and we do have a lot of primary care providers who listen to this podcast, if we're concerned, then is it pediatric neurology, the entry into this? Where do we refer? Or is it the genetics folks?

[Dr Megan Waldrop]
So here at Nationwide Children's, it can actually be a neuromuscular referral. So, we have a separate neuromuscular department. I'd say probably at most centers around the country, it's pediatric neurology.

And in rare centers, it's genetics. But we're lucky here that we have a really close relationship with our genetics department. And we have a dedicated genetic counselor in our clinic.

And so anytime anything goes to the genetics department that might be neuromuscular in nature, they immediately contact our genetic counselor. And then if it's appropriate, we just have them directly seen with us to save the families an additional visit. Yeah.

[Dr Mike Patrick]
Now, once you have the diagnosis, what are the main treatment options that are available these days?

[Dr Megan Waldrop]
So, it's getting really complicated, which I think is exciting and also frustrating, perhaps, for families. But, you know, more than approximately 10 years ago or so, we only had corticosteroids. And now we have several options, but it's very complicated about who's eligible and what's the best choice.

But we know that corticosteroids are helpful in Duchenne because they reduce the inflammation and they slow the decline. But they're nowhere near a cure. We've had several medications approved recently that are very focused and specific to what type of mutation the child has.

And that is a way of altering the gene or the genetics that are actually in the body to allow the boys to make more of the dystrophin protein. And those are called the exon skipping drugs. And these are infusions that you have to take every week.

So, they're not super fun to do. And they help a little bit. We've had another medication that applies to any boy with Duchenne.

That's another similar to the steroid, but a little bit different. And that works to also try and help with muscle repair and reduce the inflammation. But this is only available for boys over age six.

And there's, you know, some limitations with side effects on that. And then most recently, we have a gene therapy that was approved. And again, this is based on gene mutations.

So, there's a small subset of mutations that aren't eligible for this. You have to be four years old or older. You have to still be walking.

And it's a medicine, again, it helps some, but it's not the cure that we're looking for. So, there's still a lot of work that we need to do in the field. And I also want to say, I think it's very important to diagnose as early as possible.

Because like I mentioned before, I want to get these kiddos into early intervention or help me grow or therapies. So, we can really make sure that every aspect of their development is on track or as optimal as it can be.

[Dr Mike Patrick]
Yeah. I think because of the awareness of this particular condition, there were a lot of genetic breakthroughs that have occurred that have come out of the science of trying to find a cure for this. And in some cases, those things have found cures for other conditions that maybe it's easier to make the protein or something.

I don't know exactly why. But it does seem like this work has impacted far more kids and families than just those impacted by Duchenne. Would you say that's accurate?

[Dr Megan Waldrop]
Definitely. So, we have had near cures than some other conditions. And it's because their genes are smaller.

And the protein that's made from pretty much everyone who has it is very similar, or the lack of protein is very similar. The DMD gene is huge. And also, it's very variable about all the different types of proteins or limited proteins that folks make.

And so, it's not as simple as we can just give back the gene and it'll do what it needs to do. We can't give back the full gene because it's so huge. And so, people are looking at ways to see if that can actually happen, like how can we innovate in the field to make that happen.

There's also concerns about longevity. Muscles turnover constantly, right? So, we're continuing to grow and move and be active.

And if you put a gene product into a muscle cell that then kind of breaks apart and tries to reform, there's concern about does the new product that's there stay or does it go away? And so, there's lots of work trying to figure out how we can get to a point where we can give this gene therapy and it can be highly effective and it can persist for a lifetime. So those are areas that the community is still working on.

Yeah.

[Dr Mike Patrick]
And so, we still have a long way to go, but we have also come a long way, and we have impacted lots of other diseases in learning ways to manipulate genes and such. One thing I think is always important is for all of us to have some understanding of diseases that may impact people we know in our community, family, friends, and to be able to have empathy for what these folks go through. What does the day-to-day life look like for a child with Duchenne muscular dystrophy and their family, just so that we can all have some understanding of the challenges that are present?

[Dr Megan Waldrop]
Well, I mean, it can be variable, right? I've been saying this throughout the talk that we've had. So, some folks are very affected, some are less affected.

But what we do know is that over time, the muscles get weaker. And so over time, it gets harder to get around. And that means maybe needing to be in a wheelchair or a stroller part-time and then eventually going into a wheelchair full-time.

And so, families will need to get these chairs and then perhaps they have a vehicle that does not accommodate these chairs. So, there are a lot of accommodations and changes that can be very difficult to fund. And also, insurance can be very difficult to do that as well.

These boys often go to school, and some do very well in school and some need some assistance but also can do very well in school. We have several young men in our clinic who have gone to college and who are now working and leading very interesting and fulfilling lives. We've had some men in our clinic who've gone on to get married.

And so, they do need extra assistance, and life is much more challenging. But I think it's important to remember that these are boys who have unique interests and personalities and are just like anyone else in terms of their interactions and growth and ability to participate in society. And they really need our guidance to make sure that they can do that as easily as possible in this very challenging environment that sometimes is not as accommodating as it could be.

[Dr Mike Patrick]
You've talked about some of the mental health challenges that go along with this. Not only that could potentially be caused by the mutation that's causing the dystrophin protein to not be made correctly, but then also just the same sort of mental health challenges that go along with any chronic medical condition like anxiety, depression, school avoidance, all of those things. We've talked about the importance of that mental health support.

I would think it's also important to have some mental health support for the whole family, especially mom and dad. When you have a baby, you expect them to be healthy. You have visions of what their life is going to look like.

And then when things aren't necessarily going in the direction you expected, you may have anxiety and depression and all of those things yourself. And so, is there a push to kind of ask the family, like, hey, how are you guys doing?

[Dr Megan Waldrop]
We do have a psychologist in our clinic and a social worker. So, I think we try and do a very good job of checking in with everyone that's there. So, when the children are very young, there tends to be, well, even throughout the lifespan, there tends to be a lot of mom guilt because this is an X-linked condition and the moms tend to feel like it's their fault, which is not the case.

But it is something very real and something that we see all the time in our clinics. So, trying to help them not feel that way as best we can by supporting them. Also trying to encourage when we get to a point respite care, you know, if the parents are the primary caregivers, making sure that encouraging families to highlight strengths and encourage boys to pursue passions, I think is very helpful as well.

We now have a peer navigator in our clinic, and it's a young woman living with another condition, but she has to use a wheelchair and she needs significant assistance, but she's out in the world and she's working. And I think that's been very helpful for our families as well. Several parents have told me, oh my gosh, it's been such a joy to work with this woman because now I have hope for my son or daughter.

I can see that there's potential for a future. I think that's a good summary of what we try and do. We're also going to be hosting, last year and for the next few years, we're planning on hosting a family day for muscular dystrophy.

And we're trying to get our community together and host educational sessions but also host networking and kind of peer-to-peer sessions where they can just get together and talk without parents around, caregivers around, doctors around, just kind of let them meet their people.

[Dr Mike Patrick]
Yeah, that would seem like a really important thing to do, and I'm sure that a lot of good will come out of that once it gets going. What about the long-term outlook for this? Has there been much movement in terms of quality of life and then life expectancy compared to back when the telethons were on, like back in the 80s, for example?

Have we seen significant improvement and more longevity, or is this still pretty life-shortening?

[Dr Megan Waldrop]
I mean, it definitely is life-shortening still. However, I do think we've made a lot of progress. We know now that starting steroids earlier provides more benefit, and so the initiation of corticosteroids has shifted younger, and I think that has resulted in increased length of life.

We have much better care of respiratory function and cardiac function, and so now I'd say it's somewhere between, you know, 30s, 40s is, you know, where life expectancy may end. But also, I think we have so much hope, and what I hear from a lot of the young men in clinic is that they're so grateful that the boys that are just going through the clinic now will not have the experience that they had. They actually have hope and that there's potential for significantly life-changing medicines in the very near future.

We did not used to do significant cardiac interventions. I know of a couple boys with Duchenne who've received heart transplants, and so really the thought in the community has shifted in terms of this is no longer a fatal condition where there's nothing to do. There's hope.

There are things to do. Let's make sure these boys have an amazing quality of life because we're going to keep extending length of life. I really think that's how we're looking at it now.

[Dr Mike Patrick]
You know, in other conditions where we have had similar success, and I'm thinking about things like congenital heart disease and cystic fibrosis where we really do have what was once mostly childhood condition expanding into adulthood, and then the question becomes, well, where does that care go? Does it stay with the pediatric folks? Do we have specialists now who like adult congenital is now a specialty within cardiology?

We're seeing more pulmonologists that were adult pulmonologists taking care of some folks with cystic fibrosis. Is this the same sort of thing or do you guys hang on to these patients through the end of their life?

[Dr Megan Waldrop]
Yeah, that's a great question, and there's a lot of discussion right now in the field about that. So, we are trying to work on transition. However, because our clinic here was started by Dr. Jerry Mandel, who's an adult neurologist, we have been seeing adults in our clinic, and we will continue that. Additionally, because of the very complicated cardiac interventions that are happening, some of our cardiologists here are not super comfortable transitioning them to adult cardiology at adult centers. They're working on establishing relationships and educating those individuals so we can do that. So, we're working on kind of a tiered process where some of our individuals we do feel like could do the transition safely, but for those that are not in that space just yet, we plan on holding on to them.

But it does get complicated, right, when you're in a children's hospital and if you have like a 40-year-old that needs to get admitted. But I think we're all just focused on the best care, and I think educating the field, perhaps transitioning fellowships to be a little more transitional where you get both pediatric training and some adult training so we can keep helping these boys and young men as much as possible.

[Dr Mike Patrick]
Kind of in the vein of adult congenital heart where, I mean, there are specialists that are really just there for the folks who have sort of graduated into adulthood but still have that same level of expertise and training that the pediatric folks do just because they see a lot more of these kids. What further research developments are you looking forward to? So, gene therapies, definitely.

Are there any more areas of research that are ongoing and new?

[Dr Megan Waldrop]
Well, I mean, it's gene and genetic therapies, really, right? So, we need to get to the root of the problem, and we need to find a way to correct that problem. So, I think what's most exciting to all of us is really the gene and genetic therapies.

And I don't think there's going to be just one. If anyone who's listening to this knows about SMA, we know there's this great one type of gene therapy for SMA that is fantastic. But I don't think we're going to have that for Duchenne muscular dystrophy, but I think we could have several options.

And so, I talked about Exxon skipping the weekly infusions. We now have kind of what I want to call like maybe a second-generation version of that, where they only need the infusions maybe every four to six weeks, and they work so much better. In some cases, it seems like they're normalizing the creatine kinase or that muscle enzyme that leaks out when the cells break down.

So, I'm very excited about those because they're not at all anywhere near as risky as gene therapy, and they seem to be highly efficacious. But what's limiting about those is they're only for specific mutations, so it's not for everyone. So, I think we are going to have several, I hope, in the future, gene replacement or genetic therapy type options.

And I'm happy that I see one that I think will be approved soon, and I hope kind of start the influx of many more to come.

[Dr Mike Patrick]
Yeah, that would be fantastic. And it's so great that there is already prolonging the lifespan and also hope for doing even more of that in the future. Well, thank you so much for stopping by.

This has been a really fascinating conversation. And this is one of those rare diseases where we are making a difference in kids' and families' lives, and that's just so important. And of course, raising awareness.

We are going to have some links for you over in the show notes. If you head over to pediacast.org and click on the show notes for this particular episode, we'll have a link to Pediatric Neurology at Nationwide Children's Hospital, also our Muscular Dystrophy Clinic here. The MDA, or Muscular Dystrophy Association, has a lot of resources for parents that are impacted with muscular dystrophy.

But also, I think the general public, if this is something that you're interested in learning more about or volunteering in this area, getting connected with the MDA may be helpful to see what's out there and just to learn more about the condition. And then Parent Project Muscular Dystrophy, I've not heard of that one before. Is that a support group?

[Dr Megan Waldrop]
It is. I mean, it's a huge organization now, but it was started by a mother of a couple young men with Duchenne to really transform the field. So, they fund a lot of research.

They're a very large organization. They can be a great resource for families who are newly diagnosed. They have a conference, a family conference every year.

[Dr Mike Patrick]
Great. And we'll put a link to their website in the show notes as well. So be sure to check that out if you want to learn more.

Once again, Dr. Megan Waldrop, Pediatric Neurologist at Nationwide Children's Hospital. Thank you so much for stopping by and chatting with us today.

[Dr Megan Waldrop]
Thank you very much for having me.

[MUSIC]

[Dr Mike Patrick]
We are back with just enough time to say thanks once again to all of you for taking time out of your day and making PediaCast a part of it. We really do appreciate your support. Also, thanks again to our guest this week, Dr. Megan Waldrop, Pediatric Neurologist at Nationwide Children's Hospital. Thank you so much for joining us. Don't forget, you can find us wherever podcasts are found. We're in the Apple Podcast app, Spotify, iHeartRadio, Amazon Music, Audible, YouTube, and most other podcast apps for iOS and Android.

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We do turn the science up a couple notches, and we offer free continuing medical education credit for those who listen. It is category one credit, not only for physicians, but also for nurse practitioners, physician assistants, nurses, pharmacists, psychologists, social workers, and dentists. It's because Nationwide Children's is jointly accredited by all of those professional organizations that we can offer the credits you need to fulfill your state's continuing medical education requirements.

Shows and details are available at the landing site for that program, PediaCastCME.org. You can also listen wherever podcasts are found. Simply search for PediaCast CME.

And then one more podcast that I host is called FAMEcast. This one's a faculty development podcast from the Center for Faculty Advancement, Mentoring, and Engagement at The Ohio State University College of Medicine. So, if you are a teacher in academic medicine or a faculty member in any of the health sciences, then this is a podcast for you.

And you can find FAMEcast at FameCast.org, also wherever podcasts are found by searching for, you got it, FAMEcast. Thanks again for stopping by. And until next time, this is Dr. Mike saying stay safe, stay healthy, and stay involved with your kids. So long, everybody.

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