FDA Approves Previously Rejected Muscular Dystrophy Treatment
FDA Approves Previously Rejected Muscular Dystrophy Treatment
The drug Vyondys 53 has met a surrogate endpoint of protein production, but there’s no clear proof it has a clinical benefit.
FDA Approves Previously Rejected Muscular Dystrophy Treatment
FDA Approves Previously Rejected Muscular Dystrophy Treatment

The drug Vyondys 53 has met a surrogate endpoint of protein production, but there’s no clear proof it has a clinical benefit.

The drug Vyondys 53 has met a surrogate endpoint of protein production, but there’s no clear proof it has a clinical benefit.

Duchenne muscular dystrophy
Trial of Gene Therapy for Duchenne Muscular Dystrophy Put on Hold
Trial of Gene Therapy for Duchenne Muscular Dystrophy Put on Hold
Catherine Offord | Nov 12, 2019
The US Food and Drug Administration halts a study by Solid Biosciences after a patient experiences severe side effects following treatment.
Image of the Day: Dystrophin Restored
Image of the Day: Dystrophin Restored
Carolyn Wilke | Mar 21, 2019
CRISPRed heart muscle cells from humans gain the ability to make a protein missing in Duchenne muscular dystrophy.
Putting Exosomes to Work
Putting Exosomes to Work
Ruth Williams | Sep 1, 2018
Researchers identify a handy tool for tinkering with the versatile vesicles.
Infographic: Directing Exosome Traffic
Infographic: Directing Exosome Traffic
Ruth Williams | Sep 1, 2018
Researchers harness a protein to send cargo-carrying exosomes to mouse muscles.
CRISPR Treatment for Duchenne Muscular Dystrophy Helps Dogs
CRISPR Treatment for Duchenne Muscular Dystrophy Helps Dogs
Catherine Offord | Aug 31, 2018
Researchers boosted levels of the dystrophin protein to nearly normal levels in a canine model of the disease.
Infographic: Treating Duchenne Muscular Dystrophy with CRISPR
Infographic: Treating Duchenne Muscular Dystrophy with CRISPR
Sandeep Ravindran | Aug 30, 2018
The disease is caused by mutations in a single gene. Can gene editing fix the problem?
Gene Editing Could One Day Treat Muscle Disorders
Gene Editing Could One Day Treat Muscle Disorders
Sandeep Ravindran | Aug 30, 2018
Scientists race to develop CRISPR therapies that could save the lives of kids with muscle-wasting conditions.
Positive Trial Results for Experimental DMD Gene Therapy
Positive Trial Results for Experimental DMD Gene Therapy
Catherine Offord | Jun 20, 2018
Preliminary data from Sarepta Therapeutics has exceeded expectations, causing a surge in company stock prices this week.
Nonviral CRISPR Delivery a Success
Nonviral CRISPR Delivery a Success
Abby Olena | Oct 2, 2017
Researchers use gold nanoparticles to deliver CRISPR-Cas9 and correct a point mutation in a mouse model of Duchenne muscular dystrophy.