I wrote last week that I have been overwhelmed this summer by the demands of being a mom and caregiver.

I have seven children with my husband, Jason: Lexi, 25; Max, 20; Chance, 19; Rowen, 17; Charlie, 15; Mary, 11; and Callie, 4. Max, Rowen, and Charlie live with Duchenne muscular dystrophy (�鶹��). Caring for my three sons with �鶹�� has left me feeling frazzled and exhausted, especially as we’ve learned to manage Rowen’s newly broken femur.

I wish I could say that I am feeling much better this week, that I had plenty of rest and lots of help, and that I’m like a new person. While I did make some progress, when caregiving becomes stressful and the caregiver is tired, that feeling of being overwhelmed doesn’t just dissipate.

A new caregiver came over who will help one night a week with the bedtime routine, and I hope to see that expand. I also had one great night of sleep and a couple of good nights, all of which were needed and appreciated, and I had a good cry in bed one night as I said my prayers. Those tears running down my cheeks lifted the weight off my shoulders. Sometimes I don’t realize how much I am carrying until it lifts.

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Admitting my stress makes me feel guilty

When I feel overwhelmed, I tend to think it’s from caring for my sons with �鶹��, but after spending many afternoons this summer with my two young daughters, I realize it’s the combination of caring for my sons and being a mom to all my kids.

I love having a big family, and I’ve written about how it adds to the quality of Max, Rowen, and Charlie’s lives. It gives my sons many opportunities to meet people, share friends with their siblings, and get out of the house to attend sporting events and performances. Not to mention the abundance of love.

I rarely share the more stressful side of having a large family and three sons with Duchenne because it makes me feel guilty. I feel guilty when I give more of my time and attention to Max, Rowen, and Charlie. Most of the time, though, their needs demand it. They need me more.

It wasn’t always like that. When they were young and before the disease had progressed, they got to do most of what their other siblings did. I went above and beyond to make sure they got to experience things I knew they’d someday be unable to, and Lexi and Chance were there for all of it and enjoyed it just as much. I was giving them all special childhood memories together, and it didn’t seem that hard.

Those five kids have all grown up, and their interests have changed, but the two youngest girls have seen pictures and videos of all the things their older siblings got to do and expect the same, and I want to give them the same childhood magic.

I don’t regret my busy afternoons and fun activities at all, but I am much older, too. On top of that, Max, Rowen, and Charlie are no longer little, and taking care of them involves lifts, slings, wheelchairs, and hours of care each day. It makes me tired.

I would love to wake up, get my sons up and ready, and then have a little time to relax each day. Instead, my days are packed full of summer magic. Once my sons are settled, I’m off to make memories with Mary and Callie.

Admitting that it is harder this time around makes me feel better. It also makes it clear to me that scheduling quiet time for myself each afternoon doesn’t have to take away from caring for my sons or enjoying summer activities with my girls. There are 24 hours in a day, and I only need one or two each day to keep myself from feeling overwhelmed. This realization is another small step away from caregiver burnout for me.

���dzٱ�:��Muscular Dystrophy News Today is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or another qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website. The opinions expressed in this column are not those of Muscular Dystrophy News Today or its parent company, Bionews, and are intended to spark discussion about issues pertaining to muscular dystrophy.

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There’s a certain rhythm to gardening that I’ve always loved. The early mornings, the quiet work of turning soil, the satisfaction of seeing something grow because you cared for it. In my backyard, I’ve built raised beds filled with vegetables and herbs — nothing fancy, but enough to keep my hands busy and my mind at ease.

Living with Becker muscular dystrophy means I’ve had to adjust how I do a lot of things over the years. Gardening is no exception. I’ve learned to pace myself, to take breaks, to work smarter instead of harder. But there’s one thing I haven’t been able to fully control: falling.

“I tend to fall.”

It’s a simple sentence, but it carries a lot with it. For me, a fall isn’t just inconvenient — it can be frustrating, even a little frightening. The hardest part isn’t always the fall itself; it’s what comes after. Getting back up isn’t something I can do easily on my own anymore.

Chuck and his wife Sherri, raised five children and now enjoy time with their five grandchildren. (Courtesy, Chuck Vrasich)

For a long time, that meant relying on someone else to help me up. And while I’m grateful for the support, it’s not always ideal. You start to feel like you’ve lost a bit of independence, a bit of control over your own day.

That’s why something as simple as a piece of equipment can make such a big difference. I first heard about the

through my �鶹�� care team. They mentioned it in passing, as one of those resources that might be worth looking into. At the time, I wasn’t sure if I’d qualify or if it would be a complicated process. A lot of programs sound helpful until you actually try to apply.

But this one was different.

Once I gathered the basic information, the application itself took me maybe 20 minutes. That was it. No piles of paperwork, no jumping through hoops. Just straightforward questions to understand what I needed and why.

For me, that need was a lightweight floor-to-stand lift.

It’s not a big, complicated machine. In fact, that’s part of what makes it so useful. If I fall, someone can bring it over, I can position myself onto it, and it helps me get back to standing. It gives me a way to recover without feeling stuck or completely dependent on someone else’s strength.

When the lift arrived — less than a month after I applied — it felt like a small victory. Not in a dramatic, life-changing way, but in the quiet, practical sense that matters just as much. It meant I could spend time in my garden with a little more confidence. It meant that a fall didn’t have to derail my entire day.

Programs like this work because they’re built around real needs. The application asks just enough to understand your situation, and the documentation is minimal —things like a quote for the equipment you’re requesting. From there, the process moves quickly. Approvals can come within a day or two once everything is submitted, and then it’s just a matter of waiting for the equipment to arrive.

That kind of efficiency matters more than people might realize. When you’re dealing with a progressive condition, delays can mean missed opportunities to maintain your independence or prevent further complications. Having access to support that’s both practical and timely makes a real difference.

Chuck enjoys growing herbs, vegetables and flowers – but the threat of a falling and not being able to get up impacted his confidence working outside. The lift changed that. (Photo courtesy Chuck Vrasich)

For me, it comes back to those moments in the garden. Being able to focus on what I’m planting instead of worrying about what might happen if I fall. Being able to get back up and keep going. It’s a small thing, maybe, but it adds up. We don’t always think about independence in terms of equipment. We think about it as something bigger — mobility, careers, major life decisions. But sometimes, independence looks like having the right tool nearby when you need it. Sometimes it’s about making sure a setback doesn’t turn into a stopping point.

I still fall from time to time. That hasn’t changed. But what has changed is what happens next.

Now, I have a way to get back up. And that’s made all the difference.

This article was provided by our partner, the . It has been reviewed by Bionews for accuracy and relevance. The views and opinions expressed are those of the author and do not necessarily reflect the views of Bionews or Muscular Dystrophy News Today.

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Two major steps are now complete in an early clinical trial testing a one-time epigenetic therapy — a treatment designed to turn on or off a specific gene without changing the underlying DNA — for facioscapulohumeral muscular dystrophy (FSHD), a genetic disease that characteristically affects the muscles of the face, shoulders, and upper arms.

announced that its first trial testing EPI-321, which targets the underlying cause of this form of muscular dystrophy, is fully enrolled, and dosing of participants is now also done.

“Completing enrollment and dose escalation marks an important milestone for the EPI-321 program,” Amber Salzman, PhD, CEO of Epicrispr, said in a that also provided interim data from the trial.

�ճ����, which launched last year, has enrolled 12 adults with FSHD, and all participants have received a single infusion of the experimental treatment, according to Epicripr. Six participants received a low dose of 20 trillion vector genomes per kilogram of body weight (vg/kg), while the other six were given a higher dose of 40 trillion vg/kg. One vector genome is basically one particle of the epigenetic therapy.

The data shared to date showed that the FSHD treatment candidate “demonstrated a favorable safety profile and early evidence of disease modification,” according to Epicrispr. The company said it plans to share its findings at a conference later this year.

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FSHD is marked by abnormal activity of the DUX4 gene in muscle cells, because a protein of the same name is produced when it shouldn’t be. This leads to muscle weakness primarily affecting the upper parts of the body, with problems typically seen in eye and mouth movements.

EPI-321 aims to use epigenetics — molecular modifications to DNA that change whether a gene is active without altering the underlying genetic code — to turn off the overactive gene, thus addressing the root cause of the disease. This is somewhat different from traditional gene therapy, which attempts to alter the actual DNA sequence, and not just how a gene is read.

The experimental treatment delivers its payload using a viral vector, which is essentially a virus that’s been modified to deliver genetic material instead of causing infection. The vector used in EPI-321 is specifically derived from adeno-associated virus (AAV).

Trial studying effects of FSHD treatment over 5 years

Salzman noted that this is the first study testing the treatment in FSHD patients.

“We completed enrollment [in the Phase 1/2 trial] as rapidly as the study protocol allowed in this complex first-in-human AAV gene therapy trial,” Salzman said. The trial has seven locations in the U.S., Australia, and New Zealand.

Its main goal is to assess the safety profile of EPI-321 for as long as five years. The study also aims to preliminarily assess the therapy’s biological activity and clinical effects.

Epicrispr last month from three patients in the low-dose group who had been followed for six months after treatment with EPI-321. Imaging data showed all three experienced increases in lean muscle mass in the months following treatment. Individual gains ranged from approximately 0.5 to 1.3 pounds of muscle, and some individual muscles increased 15% in lean muscle volume, the company said.

These gains in muscle mass are a notable contrast to the typical progression of FSHD, in which patients usually lose muscle mass over time as the disease progresses.

In addition to this benefit, the first three patients also showed improvements in measures of strength and functionality at three months. These data “provide encouraging early evidence that EPI-321 may modify the underlying biology of FSHD,” Salzman said.

Epicrispr also said that biomarker data from these participants suggest that EPI-321 is silencing the DUX4 gene as intended. Further, per the company, the therapy’s safety profile has so far been manageable, with no serious side effects reported to date.

“We look forward to presenting additional clinical data at the World Muscle Society Annual Congress in September and completing 12-month follow-up across all participants next year,” Salzman said.

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Last month, the Purple Parade featured my story on its website. Reading my prompted me to reflect on why I continue speaking up about living with Duchenne muscular dystrophy (�鶹��) and what I hope these conversations might achieve.

The Purple Parade is Singapore’s largest disability-inclusion movement, bringing people with disabilities, their families, businesses, community organizations, and government partners together to foster a nation where everyone belongs. Being included among its advocates reminded me that sharing my experiences has always been about helping others understand what life with Duchenne is really like, not about publicity.

�鶹�� is a progressive condition that weakens my muscles over time, including those responsible for breathing and keeping my heart beating. I rely on a wheelchair, a ventilator, and caregivers throughout the day. These realities shape every decision I make, from attending medical appointments to accepting invitations to speak publicly.

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Accessibility is about dignity, not just features

The profile highlighted several aspects of my advocacy, including accessible transport, policy, service animals, and writing. Yet one idea stood out to me: dignity.

People often think accessibility begins with ramps and elevators. Those features matter because they enable people with disabilities to participate in society. But living with Duchenne has taught me that dignity goes much deeper. It’s about having the autonomy to make our own decisions, being treated with respect, and knowing our voices are not only heard but also valued.

One issue I discussed in the profile was wheelchair transport, or care transport, as it is often described in Singapore’s mainstream media. It’s something that I advocate for at Shalom Medcare, where I serve as brand ambassador.

Why wheelchair transport deserves more attention

Wheelchair transport is an essential service that receives scant attention in Singapore’s conversations around accessibility and inclusion. Although public transport has become far more accessible over the years, traveling as an advanced �鶹�� survivor remains challenging. Crowded buses and trains are unsafe for me, especially since my BiPAP ventilator is mounted behind my wheelchair.

Every trip requires my caregiver and me to bring a backup ventilator and spare parts in a separate bag, in case the machine breaks down, fails, or malfunctions outside the home. I also must look up the safest navigation routes before I leave.

Walking to the nearest bus stop or train station in Singapore’s hot and humid climate is another challenge. Although my home is only 15 minutes away, it’s on higher ground, making my commute anything but smooth due to bumpy, uneven terrain, which poses another safety risk for me.

Safe wheelchair transport allows me to attend medical appointments, spend time with my girlfriend, Amanda, and meet collaborators at Rebirth Ensemble, the disability-led accessible art studio we cofounded. It has also inspired much of my advocacy for safer, more dignified transport for people like me who are severely disabled and have complex health needs.

As I reflected on my advocacy journey so far, I also thought about my late brother, Isaac, whose life with Duchenne continues to inspire everything I do. If sharing our story helps one person better understand Duchenne or encourages one decision-maker to foster a more inclusive society, then every conversation has been worthwhile. That’s the legacy I wish to leave behind.

���dzٱ�:��Muscular Dystrophy News is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or another qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website. The opinions expressed in this column are not those of Muscular Dystrophy News or its parent company, Bionews, and are intended to spark discussion about issues pertaining to muscular dystrophy.

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This year’s  campaign has raised more than $641,000 to support for children and adolescents with muscular dystrophy and other neuromuscular diseases, and to help advance research and care for these patient populations.

Resulting from a partnership between the  (�鶹��) and , this year’s campaign has included 400 Burn Boot Camp locations, with more than 150,000 members participating in fundraising and community events across the U.S. Those interested can still donate on the .

The event has exceeded last year’s total, enabling the �鶹�� to send more than 800 children to Summer Camp at no cost to families. Ten years since its launch, Be Their Muscle has raised about $3.8 million to support �鶹��’s mission.

“This campaign is about far more than fundraising. It’s about creating life-changing experiences and core memories for children living with neuromuscular diseases,” Ruth Ann Daily, �鶹��’s chief development officer, said in a . “The Burn Boot Camp community’s generosity helps make �鶹�� Summer Camp possible for hundreds of families each year, and we’re deeply grateful for their partnership.”

Morgan Kline, who founded Burn Boot Camp with her husband, Devan Kline, and serves as its CEO, added: “Our Burn Boot Camp community continues to demonstrate the incredible impact that can happen when people come together for a greater purpose.”

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A decade of support

For 10 years, Burn Boot Camp’s support to the �鶹�� , including the first therapies approved for Duchenne muscular dystrophy and the first gene therapy for . It also helped develop new therapies for , , , and .

Besides scientific research and drug development, funds raised have helped support families through the �����Ի� , which offers the opportunity to talk with an �鶹�� specialist. Additionally, Burn Boot Camp has supported �鶹��’s and to make higher education more accessible and help young people with neuromuscular diseases find new opportunities and career paths. , which offer individuals living with neuromuscular disease and their families a chance to connect and have fun, also received support.

The partnership also strengthened �鶹��’s nationwide , a hub for clinical trials and specialized neuromuscular care, and the , a platform that connects real-world clinical insights to accelerate discoveries.

As someone who has experienced the impact of �鶹�� Summer Camp firsthand, I’m so grateful for Burn Boot Camp’s support year after year.

This year’s �鶹�� Summer Camp will host 23 in-person camp sessions and one virtual session across the country through Aug. 21. The experience aims to help build confidence and independence for children and young adults through activities such as sports, games, and arts and crafts.

During the camp, trained volunteers support daily activities and personal care, creating safe and accessible environments, while health professionals ensure that medications and treatments are taken.

“As someone who has experienced the impact of �鶹�� Summer Camp firsthand, I’m so grateful for Burn Boot Camp’s support year after year,” said Lily Sander, �鶹�� National Ambassador. “Devan, Morgan, and the entire Burn community help make it possible for kids living with neuromuscular diseases to build confidence, make friends, and create memories that last a lifetime, at no cost to families!”

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I have been involved with the annual (PPMD) conference since 2002. Back then, the conference was attended by fewer than 50 people, almost no one living with muscular dystrophy (MD), and no representatives from pharmaceutical companies. The sessions were presented by scientists who did not speak in layman’s terms. As someone with limb-girdle MD, I felt overwhelmed and personally deflated by the lack of progress in disease research.

The more involved I got in the organization, the more I became an agent for change. I talked with parents and encouraged them to bring their children to the conference. I asked PPMD leadership to let me present my story.

I spoke about my social life, my career as a music teacher, my love of travel, and my amateur astronomy hobby. I shared about my support system and explained that asking for help was necessary to maintaining my quality of life.

Over time, the conference began to include more people living with MD and highlight how we can live our best possible lives. I am honored to have played a small role in that change, which has never been more apparent than at this year’s conference.

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Putting patient voices in the spotlight

In my last column, I described my work with (PAAC), a group of adults living with Duchenne and Becker muscular dystrophy. Our biggest forward-facing event is the annual conference, held last month in Orlando, Florida. There, more than 30 PAAC members led sessions as experts on life with MD.

Several years ago, we began by simply presenting topics to one another. Now, we have hundreds of people in our sessions, including adults living with MD. I believe this is because we focus on topics that resonate with the entire community, such as mental health, goals, finances, career options, travel, and relationships.

Our efforts have proven that adults with all types of MD should be central to all neuromuscular conferences. For many years, our community has watched parents, researchers, and healthcare staff meet to discuss the latest science, care guidelines, home modifications, medical visits, and accessible transportation while those of us with MD sit and listen.

We, the , are changing this.

Our movement is growing through various organizations and platforms, including LinkedIn, Muscular Dystrophy News, PPMD, and the . Many programs now include community ambassadors, and groups like PAAC develop, plan, and lead conference sessions, host webinars and livestreams, and connect with one another to keep our expert voices at the forefront.

We speak directly to biopharmaceutical companies developing drugs and tell them not to forget the older MD population when developing clinical trials. We strengthen the global MD network by speaking with our friends at the , PPMD Italy, in the U.K., and emerging adult patient networks in countries such as Mexico and Brazil.

We, the patients, blog, record podcasts, and film Reels and videos. We stream on YouTube, TikTok, and Twitch to educate others about adult life with MD. We continue to enroll in natural history studies and keep personal journals documenting our health and well-being.

We add to the scientific record by sharing data points on our disease progression, and speak with families of newly diagnosed young people about transitioning into adulthood with MD. We remind them that the sky is not falling as long as we work together to support one another.

The best part, for me, is building friendships with people I never would have met if I didn’t have MD. This is the best family that none of us asked for. Forging lifelong bonds with a community that understands is a rich reward. I work hard to ensure everyone I meet feels the same way.

I am always recharged and refreshed after the annual PPMD conference, but this year was different. It felt like a tidal wave of change. Adults with MD are in demand as public speakers and consultants to pharmaceutical companies, and our words carry weight. People are realizing that patients should take the reins in moving the field forward.

And we are just getting started. If you are an adult living with MD, please join the Muscular Dystrophy News Forums, say hello, and share your expert knowledge. All positive change begins with one interaction, one introduction, and one comment.

Let’s do this together.

���dzٱ�:��Muscular Dystrophy News is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or another qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website. The opinions expressed in this column are not those of Muscular Dystrophy News or its parent company, Bionews, and are intended to spark discussion about issues pertaining to muscular dystrophy.

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“You have your hands full.”

I have heard that said hundreds of times over the past 25 years, and I hated hearing it. I always had a response ready: “Sure, but my heart is fuller,” I would say.

However, I’m currently in a season of life and caregiving where I appreciate that someone notices that my hands are full. That simple statement can nearly bring me to tears because most days, I am overwhelmed and rarely have enough hours to get everything done.

I have seven children with my husband, Jason: Lexi, 25; Max, 20; Chance, 19; Rowen, 17; Charlie, 15; Mary, 11; and Callie, 4. Max, Rowen, and Charlie have Duchenne muscular dystrophy (�鶹��). My sons are cared for, but laundry is never done, I am frequently rescheduling appointments, and the sink is always full of dishes when I fall asleep.

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A caregiver is not a superhero

Caregiving can be beautiful, and I have shared much of that over the years, but sometimes it is a real grind, and I do not want to shy away from that side either.

Caregivers are not superheroes. We are humans who get tired and burnt out. That does not mean we are going to quit or give up. It just means caring is a high-demand job, and even when we do it with love, there are times when it is hard.

Last Friday, Rowen broke his leg. He had just gotten home from a week at Muscular Dystrophy Association (�鶹��) and wanted to sit on the couch to watch his phone and decompress after a long drive. My husband lifted him onto the couch like he had a hundred times before, but Rowen sat on his leg, causing his femur to break.

Rowen is in extreme pain, and transfers hurt him so much that he must be coaxed to use the restroom and get out of bed. We have a brace and are using pain medications, but we don’t have a good new system down yet. I struggled the first morning I had to get Rowen ready on my own and was in tears before the end of it. I apologized to him for not being able to do it well on my own.

While I was struggling to get Rowen out of bed, Max kept asking when I was going to make him breakfast. I was ready to lose my mind because he knew what I was doing. He has broken his femur before, too, and knows the struggle, so I was frustrated that he could not put his needs aside for 15 minutes while I helped his brother.

I was in a hurry because I needed to pick up Callie from dance camp and take Mary to hitting lessons. I made Max breakfast, but not with a smile. Through all of this, Charlie was in the shower, yelling that he was ready to get out, but he had to wait until I had Rowen safely in his power wheelchair.

Everything got done, but I was rushing. I feel like I am always rushing. Too many people depend on me for needs that outnumber the hours in my day.

I have noticed that if my sons see me, they need me. I can leave them with a caregiver for two to three hours, and they do not ask the caregiver for help. However, once I get home, all three ask me for everything they should have asked of the caregiver. I am reminded of baby birds all chirping for food at the same time as soon as mom returns to the nest.

There are times when caregiving has felt like second nature for me. It was peaceful, and I was happy to support my children. I am learning, though, that caregiving is not always so peaceful. It can be demanding and stressful.

I will always be there and be what my sons need me to be, but I am not afraid to admit that sometimes it is hard.

���dzٱ�:��Muscular Dystrophy News Today is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or another qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website. The opinions expressed in this column are not those of Muscular Dystrophy News Today or its parent company, Bionews, and are intended to spark discussion about issues pertaining to muscular dystrophy.

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The U.S. Food and Drug Administration (FDA) is expected to decide by Feb. 28, 2027, whether to grant traditional approval to Amondys 45 (casimersen) and Vyondys 53 (golodirsen), two exon-skipping therapies designed to treat certain people with Duchenne muscular dystrophy (�鶹��).

Both medications are currently approved in the U.S. under the FDA’s accelerated approval pathway, which allows a therapy to be made available based on early clinical data suggesting it will likely benefit patients. As a requirement of this approval, drug developers have to run additional testing aimed at verifying clinical benefit, with long-term approval contingent on the results.

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Applications seek traditional approval

Now, — which markets both Amondys 45 and Vyondys 53 — has submitted applications asking the FDA to convert the therapies from accelerated approval to traditional approval. The FDA has agreed to review the applications and has set a target action date of Feb. 28, 2027.

“The FDA’s acceptance of these applications for review is an important step for the Duchenne community,” Louise Rodino‑Klapac, PhD, president of research and development and technical operations at Sarepta, said in a .

�鶹�� is a genetic disorder caused by mutations in the gene that encodes dystrophin, a protein that’s vital for muscle health. People with �鶹�� produce little to no functional dystrophin protein, leading to progressive muscle damage that drives disease symptoms.

The gene encoding dystrophin contains many segments, called exons. When the gene is read to produce protein, the exons are strung together to form a full protein-coding sequence, sort of like putting together many words to form a sentence. Many �鶹��-causing mutations disrupt the gene’s reading frame, resulting in a scrambled set of instructions that produces no functional protein.

The basic idea behind exon-skipping therapies for �鶹�� is to skip over certain exons so the rest of the instructions are back in frame, allowing cells to produce a shortened but functional version of the dystrophin protein that may help preserve muscle health. The FDA’s decisions to grant accelerated approval to Amondys 45 and Vyondys 53 were based on early clinical trial data indicating that these therapies increased dystrophin production in skeletal muscle in patients with amenable mutations.

ESSENCE trial helps support FDA review

Sarepta’s applications seeking conversion to traditional approval are based in large part on a Phase 3 clinical trial called The study enrolled boys with �鶹��, ages 6 to 13, with mutations amenable to exon 45 or exon 53 skipping. Participants were randomly assigned to receive the appropriate exon-skipping therapy or a placebo for about two years.

The ESSENCE trial was designed to show that treatment with exon-skipping therapies would lead to better outcomes in a test of motor function that measures how quickly patients can walk up four stairs. Sarepta announced late last year that the trial had missed its goal — although stair-climbing times were on average a bit faster with the therapies than the placebo, the difference was not statistically significant, meaning it could have been due to chance.

When it announced the results, Sarepta said the trial results were likely skewed by disruptions due to the COVID-19 pandemic, which affected roughly a quarter of participants. Analyses excluding those participants showed an average difference of 0.11 steps per second favoring the exon-skipping therapies over the placebo. This difference was also not statistically significant, but Sarepta has said the magnitude of difference is clinically meaningful. The company also highlighted that safety data from ESSENCE were in line with the therapies’ known profiles, with almost all side effects being mild or moderate.

In addition to data from ESSENCE, Sarepta’s applications also include published real-world evidence on patients treated with Amondys 45 or Vyondys 53 in real-world clinical practice.

Real-world evidence also supports review

“The submissions draw on the ESSENCE study and years of published real-world evidence, which together offer a fuller understanding of how these therapies benefit patients and change the progression of disease,” Rodino‑Klapac said. “Within Duchenne, each amenable mutation defines an ultra-rare population—a small subset of an already rare disease. Across our exon skipping therapies, more than 1,800 people worldwide have been treated, and we continue to observe preservation of muscle function and slowed disease progression. In populations this small and in a disease where damage unfolds over years, real-world experience is essential to understanding how these therapies impact the disease course.”

Pat Furlong, president and founder of the advocacy group , said the FDA’s decision to review Sarepta’s applications “reflects both the progress the Duchenne community has made over the past several years and the needs that remain, while maintaining a commitment to evaluating therapies with rigor.”

“We appreciate the FDA’s continued willingness to apply regulatory adaptability in addressing the unique challenges of rare disease drug development. … We are grateful for the FDA’s engagement with the Duchenne community and the Agency’s dedication to advancing therapeutic options through pathways adaptable to rare disease,” Furlong said.

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If you’ve read my last few columns, you’ll know that I’m struggling with a lot of new issues due to the progression of my facioscapulohumeral muscular dystrophy, along with joint issues from a head-on collision with a drunk driver years ago, and just getting old. As I’ve grown weaker and stiffer and pain levels have increased, I’ve been looking for equipment and accommodations that might help me regain some independence.

Home health services haven’t really made me more independent if I interpret that term to mean doing a task totally on my own. I’m getting help with showers, toileting, meals, dressing, and many other tasks. While I’m not doing these tasks without assistance, I’m also not depending on my wife, Wendy, or my daughter, Jill, to accomplish them. In my book, that’s a win, as it relieves some of the pressure from my primary caregivers.

I’ve also just obtained a new sit-to-stand cushion for the wheelchair I’m using around the house. My previous cushion was a “catapult” style device that we originally purchased to place on my kitchen chair. When I transitioned to a wheelchair recently, we moved the old cushion to the wheelchair to help me stand to transfer. While it worked well on the kitchen chair, it didn’t work in the wheelchair. I tended to forget I was sitting on a catapult and would lean forward to pick something up and end up on the floor.

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My new cushion, from , works on air and picks me straight up instead of pushing me forward and up. It doesn’t do anything unless I push a button, so there are no longer any surprise trips to the floor. It has a nice, 2-inch layer of foam on the top, so it’s also a huge improvement in terms of comfort when compared to the catapult.

It’s a challenge to move myself around the house in a wheelchair. One thing that has caused a huge loss of independence is the fact that I can’t get the wheelchair into the bathroom or the kitchen.

I now leave my walker just inside the bathroom door. I pull the wheelchair to the door threshold, which is too high for me to get the wheelchair over. Then I inflate my new cushion to a height that allows me to stand and use the walker to navigate in and out of the bathroom. Unfortunately, my slide/shuffle technique for navigating around with the walker isn’t very safe anymore. I’m afraid that my next fall will be in the bathroom.

Our kitchen floor is about 3/4 of an inch above the other floors in the house. When I try to take the wheelchair into the kitchen, I just spin the wheels and go nowhere. We’re now eating in the dining room, but that only solves part of the problem of being locked out of the kitchen. I’m now totally dependent on Wendy, Jill, or a home health aid to prep meals, do my old dishwashing job, make a cup of coffee, or even get a glass of water.

To solve the problem of full access to the bathroom and kitchen, I’m now in the process of trying out a power wheelchair equipped with lidar technology (short for light detection and ranging) to help me navigate the house safely. Last week, at the , I tried out a chair equipped with a fairly simple system with sensors that buzzed when you were getting close on your right, left, or behind you. I’m glad I tried it out, because  I couldn’t react fast enough to stop the chair before crashing into a wall on my left side. Since the chair didn’t “see” anything directly in front of it, I also nearly ran over Wendy.

The vendor of the chair, along with university staff, are in the process of getting another lidar system to try that’s supposedly smarter than the one I tried. I’m hoping it’ll work for me, but I’m not overly optimistic. This technology might not be ready for use by a blind guy.

I’m looking around for some type of angled piece of material to install at the bathroom and kitchen entrances that would allow me to get up and over in a manual wheelchair if the power chair option doesn’t work out. I’m hoping to get back to making a cup of coffee for myself and using the bathroom without feeling like I’m on a tightrope and fighting to keep my balance. As a person of faith, I have to believe that God has included all of this in his plans. Hopefully, that plan includes using the bathroom safely and making a cup of coffee independently!

���dzٱ�:��Muscular Dystrophy News is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or another qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website. The opinions expressed in this column are not those of Muscular Dystrophy News or its parent company, Bionews, and are intended to spark discussion about issues pertaining to muscular dystrophy.

The post The search for better accommodations for my disease progression continues appeared first on Muscular Dystrophy News.

Amid its ongoing review of the cell therapy deramiocel for people with Duchenne muscular dystrophy (�鶹��), the U.S. Food and Drug Administration (FDA) has scheduled a meeting with , its developer, to discuss the company’s resubmission of an application seeking the treatment’s approval.

That meeting — between Capricor and the FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee, or CTGTAC — will be held on July 29, according to a from the developer, which noted that the session will be available for live streaming.

A decision from the agency on whether or not to approve deramiocel for �鶹�� is expected by Aug. 22.

Capricor had previously requested the therapy’s approval, primarily based on Phase 2 clinical trial results, but the FDA last year rejected that application. In its decision, the regulatory agency stated that there was insufficient evidence of the treatment’s effectiveness. Capricor has since worked to bolster its case for approval.

“We are encouraged by the opportunity to bring Deramiocel before the Advisory Committee and engage directly with the FDA, the �鶹�� patient community, and the physicians who care for them,” said Linda Marbán, PhD, Capricor’s CEO, adding that the company is confident in its application. Additional information about the meeting is available on the company’s website, per the release.

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�鶹�� is caused by genetic mutations that result in virtually no production of dystrophin, a protein that normally helps protect muscles from damage during movement. This leads to symptoms such as progressive muscle weakness and wasting.

Cardiomyopathy, a condition in which the heart becomes enlarged and thickened, and its ability to pump blood is impaired, is the leading cause of death among people with �鶹��.

New trial data being used to seek approval of deramiocel for �鶹��

Deramiocel (CAP-1002) is a cell-based therapy that contains cardiosphere-derived cells — a population of immature heart cells with anti-inflammatory and immunomodulatory properties. It was designed to improve muscle strength and heart health in people with �鶹��.

The therapy is made from human donor hearts, and is delivered to patients via infusion. Then, inside the body, the therapeutic cells release tiny particles called exosomes that are taken up by immune cells called macrophages to drive them into a healing mode.

A since-completed Phase 2 trial dubbed tested deramiocel versus a placebo in 20 people with �鶹��. The results showed that the treatment improved heart and arm function in boys and young men at advanced stages of the disease. Benefits were also reported at four years of treatment from the , which overall found largely stable heart function and a slowed decline of arm function among the participants.

The then enrolled 106 boys and young men with �鶹��, most of whom were unable to walk (85%) and had cardiomyopathy (75%). Each was randomly assigned to receive infusions of either deramiocel or a placebo every three months for one year.

Top-line data from that trial showed that the therapy stabilized the progression of left ventricular ejection fraction — a measure of how effectively the heart pumps blood out to the body. The effects on heart function were particularly pronounced in patients with cardiomyopathy at the start of the study, who actually experienced slight improvements.

Besides the heart, deramiocel also slowed decline in arm and hand function relative to the placebo, as measured with the Performance of the Upper Limb test. The resubmitted application includes the Phase 3 trial data, according to Capricor.

We have confidence in the totality of evidence supporting Deramiocel, which has demonstrated clinically meaningful, statistically significant skeletal and cardiac benefits with a consistent safety profile, across multiple studies supporting its potential as a first-in-class therapy for Duchenne muscular dystrophy.

“Our focus remains on supporting the Agency’s review and preparing for this meeting, with the urgent needs of the �鶹�� community guiding every step, and we remain committed to bringing this therapy to the families who need it,” Marbán said.

The company noted that deramiocel achieved statistical significance on its primary goal, or endpoint, and secondary endpoints in the Phase 3 trial.

“We have confidence in the totality of evidence supporting Deramiocel, which has demonstrated clinically meaningful, statistically significant skeletal and cardiac benefits with a consistent safety profile, across multiple studies supporting its potential as a first-in-class therapy for Duchenne muscular dystrophy,” Marbán said.

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