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.
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.
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.
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.