While the last decade has brought considerable progress for patients with DMD, substantial unmet need remains. Several ...
In year two of the trial, Elevidys demonstrated statistically significant and clinically improvements across three key ...
The gene therapy improved motor functions in children with Duchenne muscular dystrophy two years after treatment ...
The gene therapy delandistrogene moxeparvovec-rokl showed clinically meaningful benefits and disease stabilization at 2 years ...
KOIN Portland on MSN9h
Vancouver family launches son’s ashes into spaceA Vancouver family is sending their son on one final journey into the stars, where his ashes will eventually transform into a ...
(RTTNews) - Sarepta Therapeutics, Inc. (SRPT) Monday reported positive topline results from Part 2 of the Phase 3 study, dubbed EMBARK, of its gene therapy Elevidys approved for the treatment of ...
Roche and Sarepta Therapeutics have shared positive top-line results from a late-stage study of Elevidys (delandistrogene ...
Donald Trump continues to make waves in biopharma; Sage rejects Biogen’s unsolicited takeover offer; the obesity space sees ...
Joshua Mercieca had a rare condition called Duchenne muscular dystrophy, which is a genetic disorder characterised by ...
NanoCas, an ultracompact nuclease, enables efficient muscle editing in NHPs using a single AAV - advancing gene editing for hard-to-target tissues.
Prophylactic cardiac treatment may prolong survival in Duchenne muscular dystrophy (DMD), but only one-quarter of individuals ...
Investor's Business Daily on MSN2h
This Drug Stock Sits On A Strong Earnings Foundation As It Tests New EntryThis pharma stock has made strong gains over the past 12 months thanks to its strong earnings. Now it is testing a fresh buy ...
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