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Duchenne muscular dystrophy

Latest Headlines

Latest Headlines

Small biotech advances on some big goals for Duchenne muscular dystrophy

Marc Blaustein never said it would be easy to develop a new drug for Duchenne muscular dystrophy. But even though he doesn't have the biggest organization, the most money or a full set of human data on key endpoints, he has been making some steady, early progress on his quest.

Prosensa gets an FDA green light for its once-failed DMD drug

After a late-stage failure and a kiss-off from partner GlaxoSmithKline, Prosensa and its innovative treatment for Duchenne muscular dystrophy seemed to be on a downward slope. But now, after a series of "positive" meetings with the FDA, the company says it plans to submit its breakthrough-designated drug for approval this year, taking advantage of the agency's accelerated pathway.

PTC soars as EU changes its tune on the DMD-treating ataluren

Reversing an earlier rejection, European regulators are recommending early approval for PTC Therapeutics' ataluren, a treatment for Duchenne muscular dystrophy, news that more than doubled the biotech's share value in premarket trading.

Sarepta bets on DMD drug approval with a $25M plant buy

After months of back and forth with regulators yoked its share price up and down, Sarepta Therapeutics is all-in on its odds of winning FDA approval for its Duchenne muscular dystrophy treatment, putting up $25 million for a manufacturing plant to produce the drug.

Erectile dysfunction drugs improve blood flow in Duchenne boys

A class of drugs used to treat erectile dysfunction could also treat blood-flow problems related to Duchenne muscular dystrophy--a bright spot in a disease field marked by repeated clinical setbacks.

Sarepta rockets up after mapping a shortcut to the FDA with DMD hopeful eteplirsen

After keeping investors on a roller coaster ride of speculation for months, Sarepta says it now plans to shoot for an accelerated approval of its Duchenne muscular dystrophy drug eteplirsen. The biotech says it will file for an early approval by the end of this year after launching a slate of new clinical trials to get the data that the FDA is looking for.

Upbeat review of PhII DMD trial earns Prosensa a big boost to shares

After the markets closed last night, Prosensa issued an upbeat self-assessment of its 48-week extension data from a Phase II study of its lead drug drisapersen for Duchenne muscular dystrophy.

Report: Father of DMD victim raises $17M for his mission to conquer disease

Patient groups are a potent force in the Duchenne muscular dystrophy field, where parents are known to take a vocal role in pushing regulators to help biotechs advance new therapies for the lethal genetic condition. Now one of those parents, J.P. Morgan vet Ilan Ganot, has reportedly been raising some serious cash to do deals on early-stage therapies that can be rushed through clinical development.

PTC slammed after EMA spurns its bid for early OK of DMD drug

Shares of PTC Therapeutics took a hit this morning, dropping about 20% after the European Medicines Agency threw its considerable weight against the biotech's application to gain a conditional approval for its Duchenne muscular dystrophy drug ataluren.

Prosensa heralds new hope for once-failed muscular dystrophy drug

Prosensa's Duchenne muscular dystrophy treatment endured a Phase III setback that led partner GlaxoSmithKline to head for the exit, but the Dutch biotech is back with a new analysis of drisapersen's data that could spell a path forward, sending its shares up 25%.