Long-term data from DEVOTE/ONWARD studies show benefits of high dose nusinersen in people living with spinal muscular atrophy (SMA)New Phase 1b data further illustrate potential of salanersen in SMA, ...

Solid Biosciences Inc. (Nasdaq: SLDB), a life sciences company developing precision genetic medicines for neuromuscular and ...

Because technology is a part of columnist Shalom Lim's daily life with Duchenne muscular dystrophy, he weighs in on the ...

A RECOMMENDATION for the HSE not to cover the cost of a drug to help children with a rare muscular disease has been blasted by families. Last year, The Irish Sun highlighted the plight of ...

Catalyst Pharmaceuticals, Inc. ("Catalyst") (Nasdaq: CPRX), a commercial-stage biopharmaceutical company focused on in-licensing, developing, and commercializing novel medicines for patients living ...

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 ...

Seven-year-old William, a Southern Oregon boy living with Duchenne muscular dystrophy, is going to get a never-before-tried surgery designed specifically for his genetic mutation. NBC5’s medical ...

On March 10, 2026, from 12:00 p.m. -- 1:30 p.m. ET, in room Key West ABCD at the Hilton Orlando, Avidity will host an industry forum lunch titled "Biomarker Advancements in Rare Neuromuscular Disease: ...

William is a seven-year-old boy from Southern Oregon living with Duchenne muscular dystrophy. Now, he is preparing to undergo a surgery that has never been tried before, a procedure designed ...

On Midday Minute for Wednesday, February 25, Sen. John Carley has been found, a South Dakotan received the Medal of Honor and a local family is looking to find a ...

The Food and Drug Administration’s vaccine chief, Dr. Vinay Prasad, will depart the agency next month, an FDA spokesperson ...