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.
Prosensa has begun the FDA submission process for its treatment for Duchenne muscular dystrophy, planning to fully file by year's end with hopes of finally winning approval for a drug that has survived a few brushes with failure.
Prosensa, one of a handful of biotechs racing to develop treatments for Duchenne muscular dystrophy, has secured $7 million from a nonprofit to accelerate its R&D efforts.
After missing a key trial goal and getting a rare second chance from EU regulators, PTC Therapeutics' ataluren is now the world's first approved therapy for Duchenne muscular dystrophy, picking up a conditional European OK as the company awaits results from a late-stage study.
The FDA's Janet Woodcock may be Sarepta CEO Chris Garabedian's favorite person today.
After Sarepta Therapeutics' former head scientist blamed his swift ouster on "serious disagreements" with CEO Chris Garabedian, the company has moved to limit its chief executive's power, according to a report, especially when it comes to meetings with the FDA over the biotech's much-scrutinized lead drug.
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.
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.
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.
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.