Duchenne muscular dystrophy

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

The US Food and Drug Administration halts a study by Solid Biosciences after a patient experiences severe side effects following treatment.

CRISPRed heart muscle cells from humans gain the ability to make a protein missing in Duchenne muscular dystrophy.

Researchers identify a handy tool for tinkering with the versatile vesicles.

Researchers harness a protein to send cargo-carrying exosomes to mouse muscles.

Researchers boosted levels of the dystrophin protein to nearly normal levels in a canine model of the disease.

The disease is caused by mutations in a single gene. Can gene editing fix the problem?

Scientists race to develop CRISPR therapies that could save the lives of kids with muscle-wasting conditions.

Preliminary data from Sarepta Therapeutics has exceeded expectations, causing a surge in company stock prices this week.

Researchers use gold nanoparticles to deliver CRISPR-Cas9 and correct a point mutation in a mouse model of Duchenne muscular dystrophy. 

Researchers restored muscle function in animals with muscular dystrophy.

Scientists use the gene-editing tool to treat animals with a rare form of congenital muscular dystrophy.

Adding RNA sequencing analysis to genomic sequencing helps scientists uncover mutations likely responsible for genetic disorders they might otherwise miss.

Using CRISPR-Cpf1 gene editing, researchers have fixed mutations that cause a form of muscular dystrophy in cultured human cardiomyocytes and a mouse model.

The agency OKs Sarepta Therapeutics’s treatment for Duchenne muscular dystrophy through its accelerated approval pathway, which requires a confirmatory clinical trial.

A federal advisory panel votes against Sarepta Therapeutics’s treatment for Duchenne muscular dystrophy.

A biotech company reports that an experimental treatment elongated its CEO’s telomeres. 

Three groups of researchers used the gene-editing method to restore a protein deficient in Duchenne muscular dystrophy.

Redirecting the gene-editing tool to modulate gene expression, researchers restore protein function in cells from a child with Duchenne muscular dystrophy.