News

Sarepta Provides Safety Update for ELEVIDYS and Initiates Steps to Strengthen Safety in Non-Ambulatory Individuals with Duchenne
Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today provided a safety ...
STAT8h
Sarepta reports second patient death after treatment with Duchenne gene therapy
Sarepta said it was halting shipments of its Duchenne gene therapy for patients who can no longer walk, following the death ...
Sarepta Reports Second Death of Patient Using Its Gene Therapy
Sarepta Therapeutics Inc. said a second patient has died of acute liver failure after being treated with its gene therapy for ...
Sarepta reports second case of liver failure death after its gene therapy treatment
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 ...
Belfast Live on MSN2h
Charity backs brave NI boy's campaign to "end cruel postcode lottery" for drug
The estimated cost that Belfast Trust has produced seems high and does not align with any other figures we have seen from ...
CHCH TV16h
‘Ari’s Army’: small town shows big love for boy with rare illness
It's a rare genetic disorder affecting less than one percent of the population, and three-year-old Ari Knowles has it.
The Post-Journal9h
Tantillo And Bell Win Italian-American Charity Golf Tournament In Playoff
After the completion of the 54th Italian-American Charity Golf Association Tournament on Saturday evening, it was time for ...
PipelineReview.com8h
Roche provides safety update on Elevidys™ gene therapy for Duchenne muscular dystrophy in non-ambulatory patients
BASEL, Switzerland I June 15, 2025 I Roche (SIX: RO, ROG; OTCQX: RHHBY) announced today new dosing restrictions, effective immediately, for ELEVIDYS™ ...