Duchenne muscular dystrophy

Latest Headlines

Latest Headlines

With a Duchenne MD patient struggling for life, little Akashi suspends study

Nonprofit-backed Akashi Therapeutics has suspended a trial of its lead drug for Duchenne muscular dystrophy after a patient on the highest dose experienced "life-threatening health issues."

FDA snow day delays review of Sarepta's Duchenne drug

The FDA is taking a snow day this Friday, abandoning scheduled plans to submit Sarepta's Duchenne muscular dystrophy drug to a review by outside experts.

UPDATED: Sarepta shares crash on a harsh FDA review of Duchenne's drug

Sarepta's quest to gain an accelerated approval for eteplirsen was subjected to some harsh scrutiny by regulators at the FDA. And the insiders concluded that much of the data submitted for an approval was a mess, with weak, inconsistent data raising serious questions about the efficacy of the drug.

FDA slaps down BioMarin's Duchenne's drug as rival nears a moment of truth

After outlining its objections to BioMarin's application for its experimental Duchenne muscular dystrophy drug drisapersen in an internal review and hearing clear objections from outside experts, the FDA has rejected the biotech's marketing application, saying it has yet to see real evidence of efficacy.

CRISPR/Cas9 trifecta: Researchers correct genetic defect that triggers Duchenne muscular dystrophy

Notch up another big score for CRISPR/Cas9 gene-editing technology. Already one of the hottest new technologies in the lab, a group of investigators at UT Southwestern used CRISPR/Cas9 to correct a genetic mutation that causes fatal cases of Duchenne muscular dystrophy.

FDA puts off its decision on BioMarin's DMD drug with Sarepta on the docket

The FDA is postponing its final decision on BioMarin's treatment for Duchenne muscular dystrophy after taking a dim view of the drug in a staff-prepared review.

BioMarin's hopes in DMD fade after FDA panel pillories its case for approval

BioMarin's odds of winning FDA approval for a Duchenne muscular dystrophy treatment got longer on Tuesday after a group of agency advisers picked apart the company's clinical evidence and raised serious concerns about its safety.

FDA takes a dim view of BioMarin's DMD drug, citing 'contradictory' data

BioMarin, preparing for a make-or-break FDA panel vote next week, got a harsh review from agency staff over its treatment for Duchenne muscular dystrophy, taken to task for "contradictory" efficacy data and "life-threatening" safety risks.

PTC's muscular dystrophy drug fails another key test with approval on the line

PTC Therapeutics' in-development treatment for Duchenne muscular dystrophy failed on its main goal in a Phase III trial. But, poring over subpopulation data, the company still believes it can make its case to global regulators.

Sarepta snags priority review status to keep pace with BioMarin in DMD race

The FDA has awarded priority review status to Sarepta Therapeutics' eteplirsen. The regulatory go sign means Sarepta is still tucked in just behind BioMarin in the race to bring a therapy for Duchenne muscular dystrophy to market, with analysts tipping the agency to make a decision on both drugs by the end of the year.