Precision begins patient enrolment for FUNCTION-DMD trial

PBGENE-DMD aims to deliver sustained muscle function improvement for DMD patients with mutations between exons 45 and 55.

Precision BioSciences Activates First Clinical Trial Site and Begins Patient Enrollment in Phase 1/2 FUNCTION-DMD Study

Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage gene editing company utilizing its novel proprietary ARCUS® platform to develop in vivo gene editing therapies for high unmet need diseases ...
GEN

DMD Patient Dies After Treatment with Sarepta Gene Therapy

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 ...

Santhera Receives Positive CHMP Opinion to Expand AGAMREE® (vamorolone) Use in Pediatric DMD Patients Aged Two and Older in the EU

Santhera Pharmaceuticals (SIX: SANN) today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion recommending ...
The American Journal of Managed Care

Patients With DMD Receiving Eteplirsen Show Prolonged Survival, Study Finds

The study provides evidence of survival benefits among patients with Duchenne muscular dystrophy (DMD) receiving eteplirsen compared with the natural history of the condition. DMD is a genetic ...
Nasdaq

SRPT Plummets on Second Patient Death After DMD Gene Therapy Infusion

Shares of Sarepta Therapeutics SRPT nosedived 42% in pre-market today after the company reported a second patient death following treatment with Elevidys, its one-shot gene therapy for Duchenne ...
GEN

StockWatch: After DMD Patient Death, Analysts Still Bullish on Sarepta

Even as investors punished Sarepta Therapeutics (NASDAQ: SRPT) after it disclosed the death of a patient treated with its Duchenne muscular dystrophy (DMD) gene therapy Elevidys ® (delandistrogene ...
The American Journal of Managed Care

DMD Gene Therapy Shows Efficacy, Safety in Patients Treated at 2 Years Old

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, ...
MedPage Today

Duchenne Muscular Dystrophy: Are Patients Getting Optimal Cardiac Medication?

Consensus directed medical therapy (CDMT) was used in 73.1% of DMD patients with moderate or severe left ventricular dysfunction, leaving nearly 30% without recommended treatment. Target doses were ...