Tag:

Duchenne muscular dystrophy

Latest Headlines

Latest Headlines

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.

Prosensa heads to the FDA with its once-failed DMD treatment

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 picks up $7M to speed up DMD development

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.

PTC wins EU approval for its DMD drug as Phase III data pend

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.

Muscular dystrophy biotechs buoyed by FDA's encouragement on accelerated OKs

The FDA's Janet Woodcock may be Sarepta CEO Chris Garabedian's favorite person today.