Duchenne Muscular Dystrophy Insights

Independent Committee Backs Elevidys Trials

April 16, 2025

Latest Research

CRISPR gene editing offers promising therapeutic possibilities for Duchenne Muscular Dystrophy (DMD) by addressing its genetic roots. This approach could lead to significant advancements in managing or potentially treating DMD. By focusing on the genetic cause, researchers aim to develop targeted therapies that improve outcomes for those affected.

The use of CRISPR in DMD represents a shift towards more precise treatments, enhancing the quality of life and potentially extending life expectancy for individuals with DMD. This emerging field holds considerable promise for effective treatments. As highlighted by Haque and Yokota (2025), this research marks an important step forward in treating 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 Study to Investigate the Safety and Biodistribution of a Single Intrathecal (IT) Injection of INS1201 in Ambulatory Males With Duchenne Muscular Dystrophy (DMD)

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)

A Study to Assess the Efficacy and Safety of Satralizumab in Duchenne Muscular Dystrophy (DMD)

DMD Voice: Qualitative Interviews With Patients and Caregivers

View the full list of clinical trials →

Community News

Harrison & DuchenneApr 12, 2025

A lot of big things are happening at once.

Harrison still isn’t feeling great and we’ve learned his scoliosis has progressed from a curve of 19 degrees to 39 degrees within a space of 12 months.

He has now been referred to a orthopaedic specialist at the Children’s Hospital.

It is probable but not 100% definite that he’ll have surgery at some point to have a rod put in his spine. Harrison is absolutely terrified at that prospect and left the room when the doctor told him as he told his sister as they waited outside he was ‘too sad’ to listen to anymore.

Fuck you Duchenne.

Muscular Dystrophy UKApr 10, 2025

⚠️ Trigger warning: Bereavement

Jack's brother Sam passed away from Duchenne muscular dystrophy so he started writing a memoir of his life and their tight bond because he wanted others "to know they're not alone".

"I've written the book I wish I'd been able to read growing up. It has always been disheartening to see such a minimal representation of wheelchair users in literature. This issue needs to be addressed, urgently.

"I want to get my book published for Sam and all those impacted by physical disabilities; I want more wheelchairs on bookshelves. People like Sam deserve to be heard."

Jack’s book is a tribute to Sam and a powerful call for visibility, understanding, and connection for families affected by complex conditions like Duchenne.

Read a snippet from his book: https://loom.ly/F4K2-q4

#Duchenne

Muscular Dystrophy AssociationApr 08, 2025

For Bryce and millions of Americans, Medicaid cuts could upend their daily lives. Basic needs such as commuting to work, showering, and even having a place to live are at risk. Medicaid is more than just health care - it’s independence. Add your voice today and tell Congress to protect Medicaid! CLICK HERE: www.mda.org/medicaid