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New Hope in Fighting Duchenne Muscular Dystrophy

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Muscular dystrophy is a genetic disorder that makes the muscles of the bodies of those affected get weaker over time. There’s no known cure, but there are treatments, and researchers continue to learn more about how to prevent and treat it. In this episode, we discuss one specific type of genetic disorder: Duchenne muscular dystrophy, and learn about a new FDA-approved gene therapy that is bringing hope to families. 

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Guests:
Omer Abdul Hamid, MD, Neuromuscular Neurologist, Nemours Children’s Hospital, Florida
Rulla Starr, Neuromuscular Program Coordinator, Nemours Children’s Hospital, Florida

Producer, Host: Carol Vassar


EPISODE 58 TRANSCRIPT

Carol Vassar, podcast host/producer:

Welcome to Well Beyond Medicine, the Nemours Children’s Health Podcast. Each week, we’ll explore anything and everything related to the 80% of child health impacts that occur outside the doctor’s office. I’m your host, Carol Vassar, and now that you are here, let’s go.

MUSIC:

Let’s go well beyond medicine.

Carol Vassar, podcast host/producer:

Muscular dystrophy is a genetic disorder that makes the muscles of the bodies of those affected get weaker over time. There is no known cure, but there are treatments, and researchers continue to learn more about how to prevent it and treat it. In this episode, we discuss one specific type of this genetic disorder, Duchenne muscular dystrophy. It all started with a voicemail left on our podcast website, Nemourswellbeyond.org.

Dr. Omer Abdul-Hamid, Nemours Children’s Health:

Hello, my name is Omar Abdul-Hamid, and I’m honored to be a neuromuscular neurologist here in central Florida. I would love to talk about the newly approved gene therapy for Duchenne muscular dystrophy.

Carol Vassar, podcast host/producer:

Nemours pediatric neurologist Dr. Omar Abdul-Hamid and his colleague, neuromuscular program coordinator Rulla Starr, join me to discuss Duchenne muscular Dystrophy in detail. Together with their team, they were the first in Florida to deliver this FDA-approved gene therapy to a young man named Jaxson. We’ll get an update on how Jaxson is doing, learn more about this gene therapy, and about the medical concerns surrounding mothers of boys with Duchenne in a few minutes. First though, the basics about Duchenne muscular dystrophy from Dr. Omar Abdul-Hamid.

Dr. Omer Abdul-Hamid, Nemours Children’s Health:

Duchenne muscular dystrophy is a devastating, progressive neuromuscular disorder that is inherited. It is inherited by a defect on the X chromosome in the DMD gene. For this reason, boys are mainly affected by this disorder. Females have two X chromosomes, so they can make up for their missing copy with the other X chromosome, whereas boys only have one X chromosome. If they have a defect on that one copy that they have, that’s it. They don’t have a backup. Most people will start to see differences even before a year of age, but these differences become more pronounced as the boy gets bigger, and usually, by age three or four, it becomes apparent that something’s really wrong.

Carol Vassar, podcast host/producer:

How is it diagnosed at the present time?

Dr. Omer Abdul-Hamid, Nemours Children’s Health:

At present, the diagnosis is based off of a molecular diagnosis For most patients. What usually brings people to care is that they’ll notice that their son is tripping and falling and not keeping up with other boys as well. They might notice that their calves are enlarged. Even in retrospect, they’ll notice their boys weren’t keeping up with other boys even before a year of age, and sometimes they will bring these concerns up with a pediatrician. Most pediatricians, myself included, we sometimes say, “Let’s give them some time to catch up. Let’s see how they do with physical therapy, and if they get better, which they often do, then maybe we don’t have to keep looking for something wrong.”

It is difficult to make the diagnosis sometimes. Once they are seen by the pediatrician, and the pediatrician notices these things and sends them to a neurologist, an astute neurologist will send for a genetic test to confirm the diagnosis, looking for a defect on that DMD gene. Sometimes a CK level is ordered, or a creatine kinase level. This is a muscle enzyme that spills out into the blood when there’s active, ongoing muscle damage. This enzyme is found in very high levels in patients with Duchenne muscular dystrophy. Even at birth, it’ll be several fold increased into the 10 thousands, and even 20 thousands and above.

Rulla Starr, Nemours Children’s Health:

The normal is below 200, so one of the things we emphasize for our local pediatricians is, if you see this tripping, falling, muscle weakness in these little boys, we recommend maybe getting a CK done. It’s a simple lab that they can order. If that flags as really high, then as a clinic, we make an effort to get those boys in really quickly.

Carol Vassar, podcast host/producer:

That’s a simple blood test. I’m curious, what is happening to the muscles physically at this point, if you think that they might have DMD?

Dr. Omer Abdul-Hamid, Nemours Children’s Health:

Yes. The DMD gene encodes for a protein called dystrophin. You can think of dystrophin as a shock absorber for the muscles. If you think about it, our muscles contract and relax all day. That contraction and relaxation causes a lot of shear forces and stress on the muscle membrane that, without that shock absorber, the muscle can’t handle that stress and becomes damaged every time the patient contracts. Every time the muscle contracts, there’s ongoing damage to that muscle membrane. This damage can be repaired, but in patients with Duchenne muscular dystrophy, the damage is so widespread that their body can’t keep up with that repair. If you or I were to go to the gym and work out our muscles, our body can keep up because it’s not every single muscle in the body that’s being damaged and ripped, and shredded. Patients with Duchenne, it’s just too much for their body to keep up with that ongoing damage, and, eventually over time, the damaged muscle gets replaced by fatty tissue and fibrous tissue, and it scars down.

Carol Vassar, podcast host/producer:

Is it painful?

Dr. Omer Abdul-Hamid, Nemours Children’s Health:

Because this process happens so slowly, it’s not typically painful. Patients will note pain in their backs, or in their muscles, but this is usually due to secondary complications of the muscle damage, contracture formation, where their joint becomes fixed at a certain angle. That can be painful, or if they get weak enough where they can’t reposition themselves in a wheelchair, then that can be painful. It’s not so much that the ongoing damage to their muscles is that painful because it’s such a long process.

Carol Vassar, podcast host/producer:

They’re simply weakened.

Dr. Omer Abdul-Hamid, Nemours Children’s Health:

Correct.

Carol Vassar, podcast host/producer:

We’re going to talk about gene therapy in a moment, but traditionally, how has DMD been treated?

Dr. Omer Abdul-Hamid, Nemours Children’s Health:

Traditionally, the mainstay of treatment has been steroids. We know that steroids help slow the progression of the disorder. They help people ambulate for longer, on average, about 18 months longer than untreated patients. We know that even once they lose ambulation, steroids are still beneficial and that they aid in heart health and respiratory muscle function. There are other treatments that have been FDA-approved for certain people with certain mutations, but these aren’t available to every single boy with Duchenne. It depends on their gene mutation whether they’d be eligible for these newer medications that are called exon-skipping medications.

Carol Vassar, podcast host/producer:

Exon skipping medications, the FDA has approved four of them, are designed to target and promote leaping over a section of genetic code to avoid specific gene mutations that are holding up the production of dystrophin and work toward producing more of the protein. That, according to the Muscular Dystrophy Association. Think of it as a molecular patch. This newest gene therapy for Duchenne is different. Rather than skipping a mutation, it delivers the missing gene to the body of Duchenne patients who meet specific parameters, including age. Dr. Abdul-Hamid.

Dr. Omer Abdul-Hamid, Nemours Children’s Health:

This treatment is called Elevidys or delandistrogene moxeparvovec. It is a delivery mechanism by which we can actually deliver the missing gene. Nature has already developed this really efficient way to inject genetic material into a cell, and that’s called a virus. We have reverse-engineered this virus to inject the genetic material that would be beneficial to this patient, the genetic material that they’re missing, and that’s the DMD gene. Unfortunately, the DMD gene is too large to fit into most of these viruses. The DMD gene is the largest gene in the body. Even half of it doesn’t fit into this virus. What they’ve had to do is engineer a modified version of that dystrophin product to make a mini dystrophin, which is less than half the normal amount. While this new engineered mini dystrophin, or micro-dystrophin, is not found in nature, it was based off of a patient who had Becker muscular dystrophy, which is a milder form of Duchenne muscular dystrophy, and that patient was ambulating into his sixties.

What’s really remarkable is that you can be missing half the gene and still have a very mild phenotype, a very minor disease. We’ve learned from that patient and others that while you could be missing a large portion of the gene, you can still have improvements in function, even with a smaller version of the gene that has the critical components.

Carol Vassar, podcast host/producer:

To be clear, when we talk about a virus, in this instance, it’s not going to give you a disease. Talk about that.

Dr. Omer Abdul-Hamid, Nemours Children’s Health:

Yes, that’s correct. This virus is just simply a shell. It’s a delivery mechanism. It has been gutted out of its normal genetic material that it would normally carry. This virus is called an AAV or an adeno-associated virus. In nature, adeno-associated viruses don’t infect humans, so they’re unable to cause disease in humans.

Carol Vassar, podcast host/producer:

Is it safe?

Dr. Omer Abdul-Hamid, Nemours Children’s Health:

It’s very safe, that’s correct. Now, that being said, this is a virus still, so the immune system, our own immune systems will attack it if it’s in a large enough quantity, which this is. We’re giving the person a very large amount of virus all at once, and the immune system is going to react to that. That’s why we give extra doses of steroids, or immune-suppressing medications, around the time of the infusion itself and for several months afterward, to try to quell that immune response.

Carol Vassar, podcast host/producer:

As a parent, I would be worried. Talk about the safety aspect of this. It is FDA-approved.

Dr. Omer Abdul-Hamid, Nemours Children’s Health:

Yes, we make sure that our parents understand that there are safety concerns that can come about from this treatment. However, they are very manageable if they’re monitored for correctly.

Rulla Starr, Nemours Children’s Health:

I also think it’s important to note that these families have been given a diagnosis for their young child that they know that there is a life expectancy that’s extremely short. A lot of these kids are only living into their twenties or early thirties, and at that point, their quality of life is really low. Yes, there is a risk, but they’re willing to risk it because they know what the outcomes are going to be if they don’t get treatment.

Carol Vassar, podcast host/producer:

We’re talking about the possibility of lengthening life and lengthening quality of life with this new gene therapy.

Dr. Omer Abdul-Hamid, Nemours Children’s Health:

That’s correct.

Carol Vassar, podcast host/producer:

All of this sounds complicated, which, complicated in medicine often means expensive. Rulla, you do the insurance claims. You are really the coordinator of all of this. Talk about the cost. Talk about how insurance companies, are they, are they not covering it? Talk about that complicated, behind machination that has to happen.

Rulla Starr, Nemours Children’s Health:

Yes, it is a very expensive drug. I believe it’s the second most expensive drug on the market. It’s millions of dollars. Consequently, insurance companies are very particular about covering it. It can be covered. Our first patient was a Medicaid patient. We are very excited. We got coverage within 24 hours. The insurance companies are excited about this treatment. They want to pay for it, but you have to qualify for it. There are very specific stipulations that go around the medications. If you know the stipulations, and you have a child that meets all the criteria, then you should be able to get treatment if it’s covered by your insurance.

Carol Vassar, podcast host/producer:

It’s interesting to hear that the first patient was a Medicaid patient. Talk about that.

Rulla Starr, Nemours Children’s Health:

Yeah, it’s really exciting. I would say 70% or more of our kids are on Medicaid. We have a huge population. Due to the diagnosis, they qualify for it. We’ve had a great partnership with Medicaid. They have really done a great job with caring for their kids, and we had no problems getting this through.

Carol Vassar, podcast host/producer:

That’s wonderful news. I want to talk about the young man. His name has been out there in the press, Jaxson, who was the first commercial patient to receive that here in the state of Florida, and it happened right here at Nemours. Dr. Abdul-Hamid, tell me about Jaxson: how he has been doing.

Dr. Omer Abdul-Hamid, Nemours Children’s Health:

Yes, Jaxson is a remarkable young man. He has done tremendously well with this treatment, thankfully. All his lab work have looked great so far. He’s not had any of the adverse side effects that we worry about. He continues to make gains in his motor function. He is running more than he was before. He is getting up from the floor easier and faster than he was before. Even within a short amount of time, within two weeks, we’ve started to notice improvements in his muscle motor function.

Carol Vassar, podcast host/producer:

What are some of the things that you look for that might be side effects of having this medication infused?

Dr. Omer Abdul-Hamid, Nemours Children’s Health:

Side effects of this medication can include toxicity to the liver, inflammation of the muscles of the heart, a drop in platelet counts or other blood counts. All these things need to be monitored closely in the laboratory, with the laboratory evaluations.

Rulla Starr, Nemours Children’s Health:

We follow up weekly, for the most part, on our labs to make sure things are on track.

Carol Vassar, podcast host/producer:

This goes on for 12 infusions. Is it once a week?

Rulla Starr, Nemours Children’s Health:

It’s one infusion, and then we follow them for 12 continuous weeks, either with labs and/or clinic appointments. At 12 weeks, we reassess and go from there.

Dr. Omer Abdul-Hamid, Nemours Children’s Health:

The actual day of infusion is an anticlimactic day. You expect this big thing to happen, but really, it’s a simple IV infusion. It’s a simple IV push. The side effects really start to come about after the infusion, either a day or two afterwards. Most patients will have some sort of vomiting, which can be managed with vomiting medications. We try to make sure that the patient doesn’t get a secondary infection because a lot of the time, we run into these side effects when a patient gets a secondary infection on top of this stressor on the body already. We ask our patients to be very mindful of where they go and what exposures they are exposed to. That’s to limit any kind of secondary infection.

Carol Vassar, podcast host/producer:

If they were going to school, maybe preschool, no preschool, staying away from siblings if the siblings are sick, parents if the parents are sick.

Dr. Omer Abdul-Hamid, Nemours Children’s Health:

That’s correct.

Carol Vassar, podcast host/producer:

It really sounds like that risk-benefit analysis done by the parents, looking at the long-term.

Rulla Starr, Nemours Children’s Health:

Yeah.

Dr. Omer Abdul-Hamid, Nemours Children’s Health:

That’s a good way to put it. It’s a risk-benefit analysis done by the parents, whether they’re willing to take the risk of the potential side effects versus the potential reward.

Rulla Starr, Nemours Children’s Health:

Yeah, I would say in the short term, it is difficult on the families. We haven’t touched on the criteria specifically, but these boys have to be between four and five years old. Four and five-year-old boys are rambunctious; they’ve got a lot of energy, and now, you have to pull them from school. We usually pull them two, three weeks prior to their infusion date. We don’t want them to get sick before they’re infused, and then they’re out of school for 12 weeks afterwards just due to viral shedding and then being immunosuppressed. We don’t want them getting sick, and we all know four and five-year-olds are always sharing germs. Now, you’ve got a mom or dad at home with their child for an extended period of time. They can’t go out to the parks, they can’t go out to shopping. They’re really in the house the whole time. We usually have families separate. If they have a mother-in-law suite or a second place they can stay, mom or dad will take the child out of the house, and they might not see their other child or their sibling for 13 to 15 weeks.

Carol Vassar, podcast host/producer:

You raise an interesting point. There is stress that happens on the family unit. Talk about that.

Rulla Starr, Nemours Children’s Health:

Well, I think they’re stressed in general as soon as they get this diagnosis and leading up to it because it’s a rare disease. Sometimes we catch it early, that’s the goal. We just had a seven-year-old who was seeing multiple specialists for years, and they never found it until they came to Nemours. Families are already stressed because they’ve gotten this diagnosis or they’ve been working through the process. They’re then excited because they have a treatment option, but then they’re stressed again because they have all these appointments, all these lab draws. It’s not fun for a four-year-old to get a blood draw every week. There’s just a lot of stress in general, but the hope that comes along with it, with this treatment, outweighs everything else.

Dr. Omer Abdul-Hamid, Nemours Children’s Health:

The other complicating factor is that about a third of our patients also have coexisting autism, which can make things a little bit more challenging. This young boy might not understand why he has to be separated from his siblings and family members and things like that. That’s another added stressor to the family.

Carol Vassar, podcast host/producer:

The routine has been broken, and that can be distressing.

Rulla Starr, Nemours Children’s Health:

And they’re not getting their other therapies.

Carol Vassar, podcast host/producer:

Exactly. Now, as a genetic disease, we often hear about newborn screening for genetic diseases. What is the current status of having DMD as part of a recommended uniform screening panel?

Dr. Omer Abdul-Hamid, Nemours Children’s Health:

Yeah, that’s a great question. There’s been a lot of effort in trying to reduce the time to diagnosis, I think we’ve touched upon that a little bit here. There hasn’t really been a reduction in the time to diagnosis in the United States. The average age of diagnosis is five to six. I would say some clinics, it’s maybe four to five, but really, it hasn’t come down despite all our efforts in educating parents, pediatricians, physical therapists, and the likes. Even though more and more providers are becoming aware of this diagnosis, and they are even aware that there’s a simple blood test that can test for this, they’re still not catching them sooner. Our average age of diagnosis hasn’t gone down. I really don’t see another avenue in which we can reduce the time to diagnosis besides newborn screening. Newborn screening is a great way to catch patients early, and right now, it is being debated on whether to add this to the recommended universal screening program, the RUSP. We should have more information on the status of that application process later on next year.

Rulla Starr, Nemours Children’s Health:

We’ve seen early diagnosis is so important in other areas. Spinal muscular atrophy, SMA, is another common neuromuscular disease, and we’re a treatment center with the newborn screening in the state of Florida for SMA. Our goal would be to also have DMD, Duchenne muscular dystrophy, on the newborn screening in the state so we can get these patients in sooner and diagnose them, and start treatment, and do the preparations so we don’t miss opportunities.

Carol Vassar, podcast host/producer:

Elevidys is not the end of the journey to treat DMD. In many ways, it’s the beginning. What’s on the horizon in terms of promising research and treatment, Dr. Abdul-Hamid?

Dr. Omer Abdul-Hamid, Nemours Children’s Health:

Yeah, there are several strategies being explored on improving dystrophin expression, and that could be through exon-skipping mechanisms. Right now, we have exon skipping for certain genotypes. However, even for those patients, yes, there is a great improvement, but there could be even a more robust response that we can see with maybe more targeted therapies that get the exon skipping ASO into the muscle better. There are several strategies currently being looked at on how to improve getting the medicine into the muscle itself. Some companies are looking on how they can attach a peptide to the current medicine, and that allows it to enter the muscle easier. Other companies are looking at, can they attach an antibody to the ASO so that it can get into the muscle easier?

There are also other dystrophin-independent strategies being explored. We have a new steroid that has just been approved by the FDA to treat this disorder, which may have less side effects, especially when it comes to bone health and linear growth. There are new treatments being explored on how to reduce the scarring in fibrosis without steroids and without the complications of steroids. Those investigational treatments are very exciting. There are other companies that are looking at gene transfer therapy, as well as gene editing therapies, in which we can actually edit the gene and make a full functional protein. Those are the things, like the CRISPR-Cas9 system, that people are excited about. All these things are very exciting. They’re in their infancy, and we hope to improve and expand upon them.

Carol Vassar, podcast host/producer:

Correct me if I’m wrong, but you have strong advocacy around Duchenne muscular dystrophy or even just muscular dystrophy in general. What would you like to see… if you had policymakers in front of you right now, legislators, payers, healthcare leaders, what would you say to them as an advocate for this community, Rulla?

Rulla Starr, Nemours Children’s Health:

Definitely, we would love to have the newborn screening. It takes resources to implement that, but we think early detection is really important. I also think that these families have a lot of complex care needs, and realistically, clinics need more support, and families need more support. When patients come to see us, they’re seeing five different disciplines all in one clinic visit, because they need it. They need physical therapy, occupational therapy, a dietician, a social worker, a clinic coordinator, a nurse, a provider. That’s one office visit. These kids also need a pulmonologist, orthopedics, imaging, genetics. We really just need funding to support these families that have already, unfortunately, progressed in the disease, and we need funding to help the families who are young, so we can keep them healthy.

Dr. Omer Abdul-Hamid, Nemours Children’s Health:

I would echo that sentiment. The other thing to add, there are certain subgroups within this population that are often forgotten, mothers of Duchenne boys.

Carol Vassar, podcast host/producer:

Talk about mothers of Duchenne boys. What’s the importance? What is their role here?

Rulla Starr, Nemours Children’s Health:

Yeah, because it is passed through the mother to the son, we do genetic testing on all of our moms to see if they are carriers for Duchenne muscular dystrophy. If they are carriers, they have an increased risk of cardiac complications. We want to make sure that they are in touch with a cardiologist that is knowledgeable about muscular dystrophy for adults. We also will test sisters because sisters could then be carriers. We haven’t touched on the sibling aspect. We have a lot of siblings that have muscular dystrophy. Mom has a baby boy, baby boy, maybe he’s developing a little bit slower, but things appear normal. She has a second baby boy. All of a sudden, the older son is diagnosed at age four or five. Well, now you have a sibling, and then they get diagnosed, and then you have a sister, and maybe she’s a carrier.

Carol Vassar, podcast host/producer:

Would she also be at higher risk for heart disease?

Rulla Starr, Nemours Children’s Health:

Yes, if you’re a carrier. Yes, the disease affects males mostly, but females also are affected.

Carol Vassar, podcast host/producer:

There’s some residual that affects moms and sisters?

Rulla Starr, Nemours Children’s Health:

And it dictates, as a woman, how you proceed in life. If I know I am a carrier of Duchenne muscular dystrophy, I’m going to think about having children in a much different way than if I’m not a carrier.

Carol Vassar, podcast host/producer:

Exactly.

Dr. Omer Abdul-Hamid, Nemours Children’s Health:

Oftentimes, when we’re speaking to carrier mothers in our clinic, we’ll ask them, “Have you gone to see your cardiologist?” Sometimes, they’ll say, “Yes, I have, but they just sent me away because I wasn’t having any symptoms,” any cardiac symptoms, chest pain, shortness of breath, anything like that. There is a lack of understanding by some cardiologists that don’t know much about the disease state and that this is an invisible disease. Sometimes we ask our moms, and they say, “No, I haven’t, because I’ve been trying to take care of my sons,” and they don’t find the time to take care of themselves.

Carol Vassar, podcast host/producer:

That’s an important part of taking care of yourself, taking care of your boys.

Dr. Omer Abdul-Hamid, Nemours Children’s Health:

Exactly. There’s also the question of whether they feel guilt or not. Some do, and they don’t want to undergo evaluations for themselves because that just plays more into their guilt.

Rulla Starr, Nemours Children’s Health:

As a mother, I personally would feel a lot of conflicting emotions about passing a genetic defect to my son, who then has a much shorter lifespan.

Dr. Omer Abdul-Hamid, Nemours Children’s Health:

I think there’s a lot of work to go still. We have work to do with other members that are affected by this disease that are often forgotten. We mentioned the carrier mothers, there are also manifesting female carriers that actually manifest symptoms early on in life. Often, adult boys with Duchenne are excluded from clinical trials. Non-ambulatory boys are excluded from clinical trials. We talked about how important newborn screening would be in identifying patients so we can improve their time to diagnosis, but we also have to figure out what to do with these newborns that have Duchenne. Right now, there’s no real consensus on what to do for them. Some people start steroids even in infancy with this disorder. Some people start exon-skipping medications if they’re eligible, with or without steroids. There’s a lot to learn even for the younger patients, the older patients.

Rulla Starr, Nemours Children’s Health:

Yeah, I would say other opportunities include adult neuromuscular care. The more and more treatment we get, the older these kids are living, which is a great thing, but then you have a lack of adult neuromuscular neurologists to send these kids to. Obviously, we’re a pediatric hospital. You can only stay with us for so long, but we struggle with finding them care elsewhere. It’s not just a neurologist they need care for. They need all the different divisions we’ve spoken about care for, and they need to be knowledgeable about how muscular dystrophy affects the lungs, affects the heart, affects everything.

Carol Vassar, podcast host/producer:

Give me some hope here. Let’s end on a positive note.

Rulla Starr, Nemours Children’s Health:

I think we’re just really excited about how quickly different therapies are coming out. I’ve only been in this space for three years, and it seems like every year we have a new treatment. I think the families of these children are wonderful advocates. They are so passionate about not only just helping their own children, but helping the community. I think that they have made significant gains for their children by advocating with different governments, and fundraising. I feel really hopeful for these families.

Carol Vassar, podcast host/producer:

Rulla Starr is a neuromuscular program coordinator at Nemours Children’s Health Florida. She was joined in conversation by pediatric neurologist Dr. Omar Abdul-Hamid.

MUSIC:

Well beyond medicine.

Carol Vassar, podcast host/producer:

Thanks to both Rulla Starr and Dr. Omar Abdul-Hamid for taking time to talk with us for this podcast episode on Duchenne Muscular Dystrophy, and thank you for listening. Both Rulla and Dr. Abdul-Hamid noted that muscular dystrophy affects every muscle in the body, and that includes the heart. We’ll have a bonus episode about how Duchenne muscular dystrophy affects the heart, featuring Nemours cardiologist Dr. Takeshi Tsuda. Look for that and all of our podcast episodes on our website, Nemourswellbeyond.org.

That’s the very same website where you can leave a voicemail with your podcast ideas, just as Dr. Abdul-Hamid did. Feel free to leave a review as well. That’s Nemourswellbeyond.org.

Our production team for this episode includes Che Parker, Susan Masucci, Lauren Teta, and Cheryl Munn. I’m Carol Vassar. Join us next time as we talk with Black leaders from across Nemours as we celebrate Black History Month. Until then, remember, we can change children’s health for good well beyond medicine.

MUSIC:

Let’s go well beyond medicine.

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Meet Today's Guests

Carol Vassar

Host
Carol Vassar is the award-winning host and producer of the Well Beyond Medicine podcast for Nemours Children’s Health. She is a communications and media professional with over three decades of experience in radio/audio production, public relations, communications, social media, and digital marketing. Audio production, writing, and singing are her passions, and podcasting is a natural extension of her experience and enthusiasm for storytelling.

Omer Abdul Hamid, MD, Pediatric Neuromuscular Neurologist, Nemours Children’s Hospital, Florida 

Guest
Dr. Hamid is a pediatric neuromuscular neurologist at Nemours Children’s Hospital, Florida. Alongside his team, he was the first in Florida to deliver an FDA-approved gene therapy for Duchenne muscular dystrophy.

Rulla Starr, Neuromuscular Program Coordinator, Nemours Children’s Hospital, Florida

Guest
Rulla Starr is a neuromuscular program coordinator at Nemours Children’s Hospital, Florida. She manages the multidisciplinary clinic, supports genetic testing, provides care coordination between specialties, and helps transition patients to adult care.

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