Duchenne muscular dystrophy

Latest Headlines

Latest Headlines

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.

Japan drugmakers steal a march on nucleic acid-based treatments

SINGAPORE-- Several Japan drugmakers are making strides toward being major pioneers in the new pharmaceuticals field of nucleic acid-based treartments. A few already have clinical trials in the works for such things as Duchenne muscular dystrophy.

Summit shoves rival Duchenne MD drug into public spotlight with $40M IPO

A biotech with scientific roots at the University of Oxford and a new approach to treating Duchenne muscular dystrophy is coming to America with a $40 million IPO.

Stanford scientists use modified RNA to rejuvenate aging cells

Scientists say that they have developed a method to use modified messenger RNA to lengthen telomeres, opening a pathway to countering the effects of aging in cells--a process that might apply to a range of diseases from DMD to diabetes and heart disease.