When Ingram became Sarepta Therapeutics’ CEO in 2017, he didn’t have a connection to muscular dystrophy, but he has developed ...

Researchers at the University of Basel have developed a gene therapy that could potentially treat a rare and currently fatal muscle disease in children. The study shows in animal models that a single ...

An El Paso mother traveled to Washington, D.C., this week to advocate for her 14-year-old son, who is living with a rare ...

Edgewise Therapeutics, Inc. (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today reported financial results for the fourth quarter and full year of 2025 and recent business ...

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, will present at the Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, taking ...

Roche’s autoimmune drug Enspryng is making solid progress as a treatment for neuromyelitis optica spectrum disorder (NMOSD).

On Midday Minute for Wednesday Sen. John Carley has been found, a South Dakotan received the Medal of Honor and a local family is looking to find a cure for ...

Why hello everyone! And welcome to a new column I like to call A Disabled Woman’s Guide. OK, it’s a working title, but you ...

After a rocky 2025, Sarepta Therapeutics’ executives admit they have work to do to bring patients back into the fold as sales of Duchenne muscular dystrophy gene therapy Elevidys continue to decline.

Last week, Eric Dane passed away from ALS, also known as Lou Gehrig’s disease. He publicly shared his diagnosis in April of ...

This award honors an emerging leader shaping the future of neuromuscular research will be presented at the 2026 MDA Clinical ...

Researchers at the University of Basel have developed a gene therapy that could potentially treat a rare and currently fatal muscle disease in ...