Topic: Duchenne muscular dystrophy (DMD)
Amid clinical holds, manufacturing concerns, safety issues and downsized IPOs, Solid Biosciences is bloodletting from its staffers.
Roche is paying more than $1 billion upfront for the ex-U.S. rights to Sarepta's Duchenne muscular dystrophy gene therapy SRP-9001.
Like its sister Duchenne drug Exondys 51, Sarepta Therapeutics’ Vyondys 53 had a roller-coaster ride toward final FDA approval.
Shares in Wave Life Sciences sunk 50% Monday morning on the news it is throwing out suvodirsen in Duchenne muscular dystrophy.
Can an unexpected Sarepta win be good news for Biogen?
Avidity Biosciences is on a roll—after inking an R&D deal with Eli Lilly and hiring a new CEO, the company is reeling in $100 million.
A new clinical hold is the latest setback for Solid Biosciences and the development of its gene therapy for Duchenne muscular dystrophy.
Sarepta’s chief Doug Ingram has come out against accusations that the FDA is biased against the company after its recent drug rejection.
The FDA rejected the New Drug Application for golodirsen, the follow-up to Exondys 51, Sarepta’s first treatment for Duchenne muscular dystrophy.
Sarepta’s stock plunged 12% on the news that a patient in a study testing its Duchenne gene therapy had been hospitalized for a serious illness.