Two interconnected themes — community and hope — dominated conversations at this year's MDA Engage symposium in Dallas.

For columnist Betty Vertin's 19-year-old son, Chance, becoming a caregiver to his brothers has benefited the entire family.

Choosing assistive equipment for a neuromuscular disease patient starts with understanding goals, environment, and needs, ...

Struggling with increasing leg weakness due to FSHD progression, columnist Robin Stemple turned to what he calls the "knee ...

Share this page with email Share this page on Facebook Share this page on X Share this page on Reddit Print Preferred Source on Google Last Friday, my girlfriend, Amanda Yip, and I celebrated the ...

A new gene therapy approach safely restored the production of emerin, the protein missing in people with X-linked Emery-Dreifuss muscular dystrophy (EDMD), in both a mouse model and human muscle cells ...

Roughly four decades into living with limb-girdle muscular dystrophy, I’ve developed numerous ways to thrive. Like many others, I believe in the power of a positive attitude. I try my best to project ...

Share this page with email Share this page on Facebook Share this page on X Share this page on Reddit Print Preferred Source on Google One problem dealing with a progressive disease like ...

On May 1, my daughter Jill graduated from the University of Pittsburgh with a master’s degree in public policy and management. She also received the department’s student achievement award and was the ...

A Phase 3 clinical trial testing delpacibart etedesiran (del-desiran), an experimental treatment for myotonic dystrophy type 1 (DM1), failed to hit its main goal. The Phase 3 HARBOR trial (NCT06411288 ...

Exon skipping is a treatment approach for people whose Duchenne muscular dystrophy is due to certain mutations in its causative gene. Exon skipping works like a molecular patch, so that the DMD gene ...

In my last column, I wrote about the increasing difficulties I’m having safely navigating my home using the “furniture surfing” technique. I wondered whether it was time that I break out the walker ...