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 ...

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 ...

When you're diagnosing a child with Duchenne muscular dystrophy, says Alexandra Bonner, MD, of Cleveland Clinic, it's important to not only determine if she's a carrier of the gene mutation that ...

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 ...

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced the ...

Muscular dystrophy is a disease caused by mutations in the dystrophin gene. The muscles of muscular dystrophy patients weaken and atrophy over time. Heart and breathing muscles may also eventually be ...

A new paper published in Gene Therapy raises serious concerns about the effectiveness of gene therapy for Duchenne muscular dystrophy (DMD), after the treatment failed to show significant benefit in a ...

Scientists have created a new gene therapy for Duchenne muscular dystrophy (DMD) that may not only help stop the disease in DMD patients, but might also help restore their damaged muscles in the ...

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 ...

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.

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

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