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 ...

Note: This story was updated Jan. 17, 2025, to clarify the company’s comments on gene therapies, which generally deliver a smaller form of dystrophin. The U.S. Food and Drug Administration (FDA) has ...

Capricor Therapeutics will resubmit its application for U.S. Food and Drug Administration (FDA) approval of its cell therapy deramiocel to treat Duchenne muscular dystrophy (DMD)-related heart disease ...

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 ...

Muscular dystrophy refers to a group of genetic diseases that cause progressive muscle weakness and loss. There are more than 30 types of muscular dystrophy, all with different causes and symptoms.