The FDA cleared Sarepta to start dosing in a first-in-human clinical trial testing the gene therapy SRP-9005 in people with ...

After a primary care visit, columnist Robin Stemple faces a battery of tests and appointments, which he calls a medical merry ...

Avidity's delpacibart etedesiran (del-desiran) was granted orphan drug status in Japan as a treatment for myotonic dystrophy ...

Columnist Shalom Lim reviews "Supervision," a play that delves into questions about privacy, dependency, and dignity.

Learning life skills to handle Duchenne muscular dystrophy can include health management, working with caregivers, and ...

Columnist Patrick Moeschen, anticipating his own historic anniversary, celebrates champions of the disability rights movement ...

Health Canada granted priority review to an application seeking approval of vamorolone, marketed in the U.S. as Agamree, for ...

The determination comes after recruitment and dosing in certain trials were paused after a young patient died due to acute ...

KER-065, a potential treatment for Duchenne muscular dystrophy (DMD) and other neuromuscular diseases, showed a good safety profile in a Phase 1 clinical trial. No serious side effects were seen in ...

Shalom Lim, who lives in Eastern Singapore, was diagnosed with Duchenne muscular dystrophy at 4 months old in 1996. He shares how caregivers can unintentionally undermine dignity and what they should ...