Fierce Pharma2h
Novartis details intrathecal Zolgensma data in bid to reach more SMA patients with gene therapy
A pair of clinical data sets gives Novartis confidence in a new formulation of the company’s gene therapy Zolgensma for the ...
MedPage Today on MSN17h
Young Patient Dies After Receiving New Gene Therapy for Duchenne Muscular Dystrophy
A young person with Duchenne muscular dystrophy died following treatment with the recently approved gene therapy ...
Sarepta says teen died after its gene therapy treatment
Sarepta Therapeutics said on Tuesday that a 16-year-old boy died from acute liver failure months after receiving the company's U.S-approved gene therapy for a rare muscular dystrophy. The patient ...
Investor's Business Daily on MSN19h
Sarepta's 27% Sell-Off Is 'Overblown,' 'Overdone' And 'Overly Bearish.' Here's Why It Happened.
Sarepta stock crashed Tuesday after a patient who received its approved gene therapy, Elevidys, died due to acute liver failure.
Le Lézard17h
EMPAVELI's Market Success Underscores Growing Demand for Complement Inhibitor Therapies | DelveInsight
DelveInsight's "EMPAVELI Market Size, Forecast, and Market Insight Report" highlights the details around EMPAVELI, a complement inhibitor indicated to treat adult patients with PNH. The report ...