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 ...

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

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).

The first gene therapy for a deadly form of muscular dystrophy received preliminary U.S. approval on Thursday despite concerns from some government scientists about the treatment's ability to help ...

Avidity Biosciences, Inc. ("Avidity") (Nasdaq: RNA), a biopharmaceutical company committed to delivering a new class of RNA therapeutics called Antibody Oligonucleotide Conjugates (AOCs™) to ...

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 ...

Experts discuss the landscape of treatment options for Duchenne muscular dystrophy, examine the benefits and risks of gene therapy and conventional steroid treatments, and emphasize the need for early ...

BELIEVE WHAT HAPPENED NEXT. FOUR YEAR OLD LUCAS LOVES A GOOD PLAYDATE AT THE PARK. HI, LUCAS. HI. IT’S SANDBOX TRUCKS TODAY, BUT HIS FAVORITE PLAYING SUPERHEROES. THIS IS HIS FAVORITE RIGHT NOW. PJ ...

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