Duchenne muscular dystrophy

Latest Headlines

Latest Headlines

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.

Sarepta and BioMarin race for an FDA tag that could be worth $350M-plus

Rivals Sarepta Therapeutics and BioMarin ​Pharmaceutical are in line to become the next recipients of an FDA coupon for a fast review, each potentially picking up a sellable asset that recently brought in $350 million on the open market.

Sarepta submits its DMD drug for FDA scrutiny, on the heels of BioMarin

Sarepta, ending a protracted process that may have cost its last CEO his job, submitted its treatment for Duchenne muscular dystrophy to the FDA, setting the stage for a possible approval early next year.

Sarepta surges after kicking off a rolling submission for its DMD drug

The FDA has given Sarepta Therapeutics the go-ahead to start a rolling submission for its Duchenne muscular dystrophy drug, eteplirsen. The green light comes 7 weeks after Sarepta parted company with its CEO Chris Garabedian--and his interim successor was quick to frame the regulatory breakthrough as the result of the subsequent shift in strategy.

Prepping for a Duchenne study, Catabasis banks $12.4M in new fundraising

Cambridge, MA-based Catabasis is once again raising cash. According to a Form D filed with the SEC yesterday, the biotech has raised $12.4 million out of a $20.4 million raise.

Summit pulls off a downsized IPO for its DMD drug

The U.K.'s Summit Therapeutics is elbowing its way into the race to treat Duchenne muscular dystrophy, pulling off a $34 million U.S. IPO to fund its research.

CRISPR project points to a new approach on Duchenne muscular dystrophy

Investigators at Duke have turned to a new gene-editing tool to come up with a new approach to Duchenne muscular dystrophy that theoretically could apply to a majority of the boys afflicted with the fatal rare disease.