Duchenne Muscular Dystrophy Insights

This week's must-know community updates, latest research & events

March 19, 2025

Introduction to Gene Therapy - out now!

We're thrilled to announce the launch of the first guide in our gene & cell therapy series.

Read it for free here:

www.allmyhealth.io/reports

Stay tuned for next week when we will launch the next report in the series.

Would you like to sponsor the next report in the series?

Enquire here

Latest Research

In a recent case report by Weerkamp et al. (2025), two adolescents with Duchenne Muscular Dystrophy (DMD) who also have neuropsychiatric and neurocognitive conditions were successfully started on Nocturnal Non-Invasive Ventilation (NIV) at home. One of the adolescents has Autism Spectrum Disorder, while the other has Attention Deficit Hyperactivity Disorder, obsessive thinking, and anxiety. The report highlights the effectiveness of a multidisciplinary approach that includes medical, psychological, and psychopharmacological strategies to address the challenges of implementing NIV in patients with such comorbidities.

The key takeaways from these cases are the importance of early care planning, accurate psychoeducation, careful use of psychopharmacological interventions, and involving both the patient and their parents. This study is the first to describe the home initiation of NIV in adolescents with DMD and additional neuropsychiatric and neurocognitive disorders, suggesting that with the right support, home implementation of NIV can be a viable option for individuals with DMD.

Clinical Trials

This is a list of upcoming or ongoing clinical trials that are actively recruiting and have been listed or updated in the last two weeks:

A Gene Delivery Study to Evaluate the Safety and Expression of Delandistrogene Moxeparvovec in Participants Under the Age of Four With Duchenne Muscular Dystrophy (DMD)

AFFINITY DUCHENNE: RGX-202 Gene Therapy in Participants with Duchenne Muscular Dystrophy (DMD)

Gamified Occupational Therapy for Adolescents with Duchenne Muscular Dystrophy

Registry Study to Observe Long-term Safety of Vamorolone (AGAMREE®) in Patients with Duchenne Muscular Dystrophy.

View the full list of clinical trials →

Community News

Parent Project Muscular DystrophyMar 18, 2025

We are deeply saddened by the loss of a young member of our community following treatment with ELEVIDYS. According to Sarepta, the individual with Duchenne received ELEVIDYS and later suffered from complications of acute liver failure, representing the first reported case of acute liver failure resulting in death following treatment with ELEVIDYS.

Our hearts ache for this family and their loved ones. We recognize the courage it takes for families to pursue new treatment options in the fight against Duchenne, and this tragic loss highlights the urgent need to fully understand the risks associated with gene therapy in our current therapeutic landscape to ensure patient safety.

Muscular Dystrophy UKMar 18, 2025

Over half of people with muscle wasting and weakening conditions receive Personal Independence Payments (PIP). It's a lifeline. We’re very disappointed the UK Government will proceed with cuts that leave us more financially insecure, affect our health and quality of life. It will damage our ability to work, not improve it.

Many people living with muscle wasting and weakening conditions do work. With the right support, more could work. But these short-term cuts and pressure to put more people into work who currently don't or can't, will have lasting consequences.

We know the welfare system needs a significant overhaul. But wanting to cut costs is the wrong way to go. Many people today will be very concerned the UK Government has decided to cut PIP and alarmed and worried about the changes.

In the next few days, we’ll be looking at the details of the consultation to better understand how these will impact people with muscle wasting and weakening conditions. We’ll share more details as well as how you can get involved.

We will also:

Work with other disability organisations to oppose cuts to these vital benefits and make the case for reform.

Respond to today’s consultation to share the concerns and needs of people with muscle wasting and weakening conditions.

If you have any concerns, we’re here for you. Call our helpline 0800 652 6352 or email info@musculardystrophyuk.org

Muscular Dystrophy AssociationMar 16, 2025

Today, March 16, on what would have been Jerry Lewis’ birthday, we honor his decades-long dedication to the Muscular Dystrophy Association and the impact he made that still inspires us today. As we celebrate MDA's 75th anniversary, we reflect on the legacy Jerry helped build—from the countless millions raised through the iconic telethon to the spotlight he shone on our community. With his friendship with MDA founder Paul Cohen, Jerry ignited a movement, bringing together celebrities and supporters from around the globe to fuel groundbreaking research, MDA Care Centers, resources, MDA Summer Camp, and advocacy efforts that changed lives. The momentum he started is alive today in the treatments and hope we see for families living with muscular dystrophy, ALS, and over 300 related neuromuscular diseases. We are forever grateful for his belief in our mission and the love he poured into this community. 💙💛🎂

Together, we honor his memory and continue his work—because every family deserves strength, care, and hope

Continue the momentum and donate at https://www.facebook.com/donate/2871114256395840/?fundraiser_source=external_url today!