The estimated cost that Belfast Trust has produced seems high and does not align with any other figures we have seen from ...

Sarepta Therapeutics Inc. said a second patient has died of acute liver failure after being treated with its gene therapy for ...

Sarepta said it was halting shipments of its Duchenne gene therapy for patients who can no longer walk, following the death ...

BASEL, Switzerland I June 15, 2025 I Roche (SIX: RO, ROG; OTCQX: RHHBY) announced today new dosing restrictions, effective immediately, for ELEVIDYS™ ...

Sarepta Therapeutics on Sunday said there had been a second reported case of acute liver failure resulting in death after a patient received the company's gene therapy for a rare form of muscular ...

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today provided a safety update regarding ELEVIDYS (delandistrogene moxeparvovec-rokl), the only ...

Eric, a beloved brother and cousin, peacefully passed away on May 25, 2025, after bravely facing the challenges of Duchenne ...

The findings support the clinical benefits of delandistrogene moxeparvovec in patients with Duchenne muscular dystrophy (DMD) ...

Participants with Duchenne muscular dystrophy (DMD) who received RGX-202 demonstrated functional improvement and better ...

Capricor Therapeutics' rating was downgraded amid FDA inspection risks, regulatory uncertainty, and potential delays in ...

Investigators found that making a few changes to a common patient-reported outcomes assessment improved its validity in patients with Duchenne muscular dystrophy.

U.S. FDA successfully completed Pre-License Inspection; Company expects facility will meet all requirements to support licensure Advisory Committee meeting scheduled for July 30, 2025 Mid-cycle ...