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. 
Dogs with Duchenne Treated with Gene Therapy
Dogs with Duchenne Treated with Gene Therapy
Diana Kwon | Jul 25, 2017
Researchers restored muscle function in animals with muscular dystrophy.
CRISPR Restores Muscle Function in Mice
CRISPR Restores Muscle Function in Mice
Diana Kwon | Jul 17, 2017
Scientists use the gene-editing tool to treat animals with a rare form of congenital muscular dystrophy.
RNA-Seq Reveals Previously Hidden, Genetic Disorder–Causing Mutations
RNA-Seq Reveals Previously Hidden, Genetic Disorder–Causing Mutations
Anna Azvolinsky | Apr 19, 2017
Adding RNA sequencing analysis to genomic sequencing helps scientists uncover mutations likely responsible for genetic disorders they might otherwise miss.
CRISPR Corrects Duchenne-Causing Mutations
CRISPR Corrects Duchenne-Causing Mutations
Anna Azvolinsky | Apr 12, 2017
Using CRISPR-Cpf1 gene editing, researchers have fixed mutations that cause a form of muscular dystrophy in cultured human cardiomyocytes and a mouse model.
Conditional FDA Approval for Fatal-Disease Drug
Conditional FDA Approval for Fatal-Disease Drug
Tracy Vence | Sep 19, 2016
The agency OKs Sarepta Therapeutics’s treatment for Duchenne muscular dystrophy through its accelerated approval pathway, which requires a confirmatory clinical trial.
Another Fatal-Disease Drug in Limbo
Another Fatal-Disease Drug in Limbo
Jef Akst | Apr 26, 2016
A federal advisory panel votes against Sarepta Therapeutics’s treatment for Duchenne muscular dystrophy.
First Data from Anti-Aging Gene Therapy
First Data from Anti-Aging Gene Therapy
Kerry Grens | Apr 25, 2016
A biotech company reports that an experimental treatment elongated its CEO’s telomeres. 
CRISPR Improves Disease in Adult Mice
CRISPR Improves Disease in Adult Mice
Kerry Grens | Jan 4, 2016
Three groups of researchers used the gene-editing method to restore a protein deficient in Duchenne muscular dystrophy.
CRISPR Therapy in a Dish
CRISPR Therapy in a Dish
Kerry Grens | Dec 8, 2015
Redirecting the gene-editing tool to modulate gene expression, researchers restore protein function in cells from a child with Duchenne muscular dystrophy.
Fatal-Disease Drug in Limbo
Fatal-Disease Drug in Limbo
Jef Akst | Nov 25, 2015
A panel of experts advised the US Food and Drug Administration that BioMarin Pharmaceutical has not demonstrated efficacy of its new drug for Duchenne muscular dystrophy.