The result comes a few months after the only FDA-approved gene therapy for DMD – Roche/Sarepta’s Elevidys (delandistrogene moxeparvovec) – failed to meet its primary objective in a phase 3 ...

The importance of Pfizer’s candidate has been heightened in the minds of the DMD community after the only FDA-approved gene therapy for DMD, Roche/Sarepta’s Elevidys (delandistrogene ...

Organon (NYSE:OGN) announced Tuesday the acquisition of regulatory and commercial rights linked to Tofidence, a biosimilar ...

Regenxbio is ranked Strong Buy due to low EV, strong partnerships, royalty income, bullish trends, and buyout potential.

While there is some uncertainty going on regarding the company’s future due to its product Elevidys’ alleged involvement in a fatal acute liver case, Mizuho views the selloff in shares of the ...

Digital therapeutics coupled with drugs has the potential to transform health care and change the way patients are treated, Anthony Costello, CEO of Medidata Solutions Inc., told BioWorld. Medidata ...

The 616 shareholders in attendance, who represented 77.03% of the total Roche Holding, Comcast, Eaton Corp and National Presto Industries are included in this Analyst Blog. Today's Research Daily ...

Sanofi SA gained U.S. FDA approval for fitusiran as a first-in-class siRNA therapy for hemophilia. Branded Qfitlia, the antithrombin-lowering therapy is indicated for use as a prophylactic treatment ...

The sector was also shaken by the death of a patient who was dosed with Sarepta Therapeutic’s DMD gene therapy Elevidys (delandistrogene moxeparvovec), after experiencing acute liver failure.

which Leerink Partners’ Joseph Schwartz noted was greater than Sarepta Therapeutics showed with its Elevidys gene therapy, which the FDA approved in 2023. But perhaps more notably, Wave touted ...