Panelists discuss how data from MDA 2025 demonstrate that delandistrogene moxeparvovec (gene therapy for DMD) shows ...

Bacteria carry countless hidden treasures in their DNA—fragments that could hold the key to new medicines. But how do you ...

A second patient has died following treatment with Sarepta Therapeutics’ Elevidys, raising more doubts about the Duchenne muscular dystrophy (DMD) gene therapy’s safety profile. | A second patient has ...

A second case of acute liver failure resulting in death has been reported in nonambulatory DMD patients treated with Elevidys.

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 ...

Sarepta Therapeutics (NASDAQ:SRPT) reported a second fatality from acute liver failure in a patient treated with its gene ...

Prime editing is an advanced gene editing technology that enables precise DNA corrections without introducing double-stranded ...

DMD gene therapy faces challenges in safety, efficacy, and long-term outcomes, despite FDA approval of delandistrogene moxeparvovec. Future strategies focus on novel AAV capsids, dual/triple ...

PBGENE-DMD is the first in vivo gene editing program that has the potential to transform the treatment paradigm and deliver durable functional improvement for most patients, as up to 60% of those ...

Scientists present current evidence for a new gene therapy for Duchenne muscular dystrophy called delandistrogene moxeparvovec. AAN Evidence in Focus articles highlight the strength of the current ...

The molecular diagnosis of DMD was established using MLPA at the National Research ... The deletion included the PARK2 gene but the exact location of the breakpoints was unknown. The boy's father did ...

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 ...