Sarepta Therapeutics said it will update its prescribing information for Elevidys ® (delandistrogene moxeparvovec-rokl) after acknowledging the sudden death of a patient with Duchenne muscular ...

Shares of Cambridge-based Sarepta rose about 35 percent in trading Wednesday after the company presented preliminary data on ...

Panelists discuss how the pooled 3-year data for delandistrogene moxeparvovec show sustained motor function stabilization and less deterioration compared with natural history, providing confidence in ...

Dyne Therapeutics’ experimental treatment for Duchenne muscular dystrophy (DMD) showed promise in new data from a small Phase 1/2 trial dubbed DELIVER, the pharma announced this week. The drug, ...

Add Yahoo as a preferred source to see more of our stories on Google. Agamree secured approvals from various nations for Duchenne muscular dystrophy treatment. Credit: MY STOCKERS/Shutterstock.com.

The patient in this case had suffered acute liver failure following the Elevidys infusion. Sarepta Therapeutics announced that a patient with Duchenne muscular dystrophy (DMD) who received Elevidys ...

The update comes as the therapy’s regulatory application awaits a decision from the U.S. Food and Drug Administration later ...

The last time I wrote about Sarepta Therapeutics, Inc. (SRPT) it was in an article entitled "Sarepta Therapeutics: BLA Filing of SRP-9003 For LGMD Is The Next Step." In this article, I noted that the ...

TORONTO--(BUSINESS WIRE)--Satellos Bioscience Inc. (“Satellos” or the “Company”) (TSX: MSCL, OTCQB: MSCLF), a public biotech company developing new small molecule therapeutic approaches to improve the ...

Delandistrogene moxeparvovec, a gene therapy for Duchenne muscular dystrophy (DMD), showed high dystrophin expression and a favorable safety profile in young patients. Based on western blot testing, ...

Eight therapies are approved for Duchenne muscular dystrophy (DMD) in the U.S., and all have been approved since 2016. "It took 30 years from the discovery of the gene associated with Duchenne ...

REGENXBIO Inc. (RGNX) has made remarkable progress in being able to advance its pipeline with various types of gene therapies. The last time I wrote about this article it was in a Seeking Alpha ...