It's been nearly two years since Elevidys, a gene therapy for Duchenne MD developed by Sarepta Therapeutics, was approved by ...

Columnist Patrick Moeschen highlights some key federal programs and pieces of legislation in the history of muscular ...

The FDA cleared Sarepta to start dosing in a first-in-human clinical trial testing the gene therapy SRP-9005 in people with LGMD type 2C.

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

I have friends and family who seem to be living on a medical merry-go-round. Every day they go to doctor’s appointments, have medical tests, or go through therapy. Their lives revolve around their ...

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

Preventive treatment with standard heart medications — when given before the onset of cardiac troubles — extends survival among males with Duchenne muscular dystrophy (DMD), according to data from the ...

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