The University of Alabama at Birmingham (UAB) has launched an expanded access program to make the experimental exon-skipping therapy delpacibart zotadirsen (del-zota) available to qualifying patients ...

Columnist Robin Stemple wasn't thrilled with the idea of respite care, but it solves scheduling challenges he has with family ...

Ongoing steroid treatment was linked to slower lung-function decline and later breathing support in older teens and adults ...

FDA advisers voted 9-3 that available evidence did not support deramiocel as a heart therapy for Duchenne muscular dystrophy.

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 The next Clinical & Scientific Conference hosted by the Muscular ...

Columnist Betty Vertin shares big news about a bigger house her family is buying that will better accommodate her sons with Duchenne MD.

“Take him home and give him a good life.” This was the main advice given to Jessica and Chris Curran by a genetic counselor after their son, Conner, was diagnosed with Duchenne muscular dystrophy (DMD ...

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 ...

While Duchenne muscular dystrophy (DMD) is one of the most common types of this group of muscle-wasting disorders, affecting about 1 in 3,500 male births, each patient’s journey with this form of ...

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 ...