Duchenne Muscular Dystrophy Insights

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

March 05, 2025

HealthSpotlight is now AllMyHealth!

Read all about our exciting merger here.

Apply now: feature your voice in our cell & gene therapy series

Cell and gene therapies are transforming rare disease treatment, offering new hope where options were once limited. But how do they work, and what choices exist for your community?

We're launching a special content series to help our 15k+ readers navigate this evolving landscape – and we need your voice!

Be part of the conversation, whether you are:

• A patient or carer

• A patient support group

• Developing cell & gene therapies

• Administering cell & gene therapies

📅 Enquiries close: March 12

Let's educate, advocate and drive change together.

Latest Research

PepGen has decided to put a temporary hold on a Phase 2 clinical trial for its experimental Duchenne muscular dystrophy drug, which is designed to skip exon 51, a specific segment of genetic code. According to Wu (2025), this pause is a precautionary measure as the company awaits safety data from a separate trial. No participants have yet been enrolled in the study, which indicates that the decision was made before the trial's initiation.

The delay underscores the importance of safety in the development of new therapies, especially when dealing with complex genetic disorders. The outcome of the safety data from the other trial will likely inform the future of this Phase 2 study, guiding whether and how it will proceed. Wu's report highlights the careful steps companies must take in the clinical trial process to ensure patient safety and the integrity of the study results.

Community News

Muscular Dystrophy UKFeb 26, 2025

“My biggest piece of advice to parents who have young children with a muscle wasting condition is to find something they enjoy doing and nurture that passion.” 😀

Ed, Lorna's son, was diagnosed with Duchenne muscular dystrophy at eight years old. He could no longer walk at aged 12.

“This was a really hard decline for him, and us as parents, to accept. But finding powerchair football changed all that,” Lorna adds. ⚽

Now 30, Ed has competed at the highest levels, including the Premier League and WFA Cup, and even represents England.

Lorna believes that supporting Ed’s passion has been positive for his mental well-being. His growing collection of trophies is proof of his talent, drive, and love for the sport.

Read the full story: https://loom.ly/ji1_x0U

Muscular Dystrophy CanadaFeb 28, 2025

Today is Rare Disease Day – a day dedicated to raising awareness and advocating for Canadians living with a rare condition.

For Gus Campbell and his family, the journey to a diagnosis wasn’t easy. But through it all, one thing became clear: no matter how rare the condition, no one should feel alone.

This year, we’re sharing Gus’s story to highlight the importance of belonging and support within the neuromuscular community. Stay tuned.

Parent Project Muscular DystrophyFeb 28, 2025

Today is #RareDiseaseDay, a day to give voice to 300 million people living with rare diseases and their families, through education and advocacy. This year, PPMD participated in #RareDC2025 on Capitol Hill, hosted by the Rare Disease Legislative Advocates (a program of the EveryLife Foundation for Rare Diseases). This multi-day event empowers participants to learn key policies affecting the rare disease community and share their stories with Members of Congress.

PPMD Advocates from across the country will be heading to Washington, DC in March for PPMD’s annual Advocacy Conference. Learn more and register today to raise your voice in the fight to #EndDuchenne:https://donate.parentprojectmd.org/event/ppmd-advocacy-conference-2025/e648216