Duchenne Families Concerns

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Sarepta's Duchenne Therapy Sparked Fears

Sarepta Therapeutics says it will pause shipments of Duchenne gene therapy

Shipments will halt by close of business Tuesday evening, the company said. Sarepta had initially rejected the agency’s request, which was issued Friday.

Bloomberg on MSN · 4h

Sarepta’s Duchenne Therapy Sparked Fears Even Before FDA’s Order

Reports of troubles linked to Sarepta’s Elevidys treatment for Duchenne muscular dystrophy had been popping up on social media for months — beyond the two deaths the company had already disclosed — but there was little information from the company or the health regulators that widened availability of the drug over objections from its own staff,

BioPharma Dive · 1d

5 questions on Sarepta, the FDA and a Duchenne gene therapy crisis

A standoff over Elevidys could have major consequences for Duchenne patients, gene therapy companies and the perception of the FDA.

Sarepta Therapeutics crisis is huge blow to Duchenne families, company

Sarepta rebuffed a call from the Food and Drug Administration to halt all shipments of its gene therapy for Duchenne muscular ...

21hon MSN

Sarepta’s Elevidys Halted at Children’s Hospital Los Angeles

Children’s Hospital Los Angeles paused the use of Sarepta Therapeutics Inc.’s gene therapy for Duchenne muscular dystrophy in the wake of rising safety concerns about liver toxicity, adding to ...

WBUR5y

Families Fought For A New Drug's Approval. Now They Fear ... - WBUR

ICER is listening to concerns like McSherry's, and Pearson says feedback from the Duchenne patient community is being incorporated into the organization's work.

Sarepta Therapeutics Stock Plunges Over 30% In Friday Pre-Market: What's Going On

Shares of Sarepta Therapeutics (NASDAQ: SRPT) plunged 26.85% in Friday pre-market after a clinical trial participant died ...

STAT9mon

Dispute over Duchenne treatment highlights thorny access issues

Finally, it seemed, Sarah Jenssen would have a chance to get a treatment that her family hoped could help her. A gene therapy for Sarah’s condition, Duchenne muscular dystrophy, won a wide ...

CNN8y

Duchenne muscular dystrophy: Families fought for new drug | CNN

Jenn McNary’s two sons have a rare disease, Duchenne muscular dystrophy. The FDA recently approved a controversial drug that could improve their lives.

Arizona Daily Star1mon

Gene therapy for Duchenne muscular dystrophy provides hope, raises concerns

“With the benefit of newborn screening, the next generation of families will have even more time to evaluate, pursue and access FDA-approved Duchenne therapies while avoiding extensive surgeries ...

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