Durham-based Precision BioSciences, a gene-editing company, has received regulatory clearance to begin a clinical study of its potential treatment for Duchenne muscular dystrophy (DMD), a rare ...

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 ...

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced the official opening of Route 79, The Duchenne Scholarship Program for the ...

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: ...

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 ...

ELMIRA, N.Y. (WETM)— Last week, Eric Dane passed away from ALS, also known as Lou Gehrig’s disease. He publicly shared his ...

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

Sarepta Therapeutics CEO Doug Ingram announced Wednesday that he will retire by the end of 2026, citing a “shocking and ...

On an earnings call, Ingram said he was leaving because his wife and son were recently diagnosed with myotonic dystrophy.

The Enquirer and United Way of Greater Cincinnati's 39th annual Wish List campaign raised $148,983 for local families.