Duchenne Muscular Dystrophy Insights

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

March 13, 2025

New This Week

Exciting news! There is now a new section featuring clinical trials that are actively recruiting patients. This highly requested addition gives you direct access to cutting-edge research opportunities that could shape the future of treatment.

Stay informed about potentially life-changing studies and let us know what you think by replying to this email!

Latest Research

In a recent case report by Gerges et al. (2025), two previously unknown gene variants associated with Duchenne muscular dystrophy were identified. The patient, who exhibited symptoms of a dystrophinopathy—a condition related to a dysfunctional dystrophin protein—underwent genetic analysis which revealed these novel mutations in the dystrophin gene.

The discovery of these mutations is significant as it expands the understanding of the genetic diversity and complexity of dystrophinopathies. The findings underscore the importance of genetic testing in diagnosing and managing these disorders. Gerges et al. emphasize that identifying such mutations can aid in the development of targeted therapies and improve prognostic assessments for affected individuals.

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:

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.

Defining Endpoints in Becker Muscular Dystrophy

View the full list of clinical trials →

Community News

Muscular Dystrophy UKMar 07, 2025

📢 New development in gene therapy for muscle wasting conditions!

Researchers have developed a new way to deliver the instructions needed to make proteins, which are often missing or don’t work correctly in muscle wasting conditions. 🧬

Gene therapies have been developed to deliver these instructions. But the challenge for many muscle wasting conditions is that the instructions can be huge! This makes it difficult to get them to the body’s cells - where they are needed.

Now, researchers have found a way to overcome this obstacle. 🧬✨ Introducing StitchR: a method that splits the instructions into smaller parts and then stitches them back together once they reach the muscle cells!

In experiments, the approach successfully led to the production of proteins like dysferlin and dystrophin in animal models, leading to muscle improvements. While this is still early research, this method could open the door to new, more effective gene therapies for muscle wasting diseases. 🚀

Read more: https://loom.ly/NrglHAg

Parent Project Muscular DystrophyMar 10, 2025

We are on the ground in Washington, DC, for PPMD’s 2025 Advocacy Conference, getting ready to head to Capitol Hill to urge members of Congress to continue making #Duchenne and #Becker patient care, public health, and research a top priority.

Today kicks off with a panel discussion about what’s going on in Washington, featuring experts to talk about NIH, DoD, Congress, and federal agencies. Thank you to our speakers: Dave Zook, Nick Manetto, Joel Wood, and John Porter. And thank you to our PPMD Advocates, both in DC and at home around the country, for sharing your powerful stories with lawmakers and making success in our community possible. #EndDuchenne#PPMDAdvocacy

Muscular Dystrophy AssociationMar 11, 2025

For Cassidy Nilles, diagnosed with Limb-Girdle muscular dystrophy (LGMD) type 2J, she faced the challenge of giving up her career as a hairstylist and navigating life as a single mom. Through struggles with depression and unhealthy coping mechanisms, Cassidy found strength in her daughter, Capri, and made the courageous decision to embrace sobriety. Now, she’s celebrating over a year of clean and healthy living—prioritizing wellness, mindful choices, and being fully present for her daughter.

Read Cassidy’s powerful journey of resilience and transformation on the MDA Quest Blog: https://mdaquest.org/mda-ambassador-guest-blog-finding-my-way-to-a-sober-and-healthy-way-of-living/