gene therapy duchenne muscular dystrophy

Order byBest matchMost fresh

FDA asks Sarepta to stop shipping gene therapy

Sarepta’s Risky Gamble: Gene Therapy Stays on Market Despite FDA Request

At the heart of the controversy is a question many patients and families are asking: Should a drug that might save lives also be allowed to risk them?

· 1d · on MSN

Gene therapy maker Sarepta tells FDA it won’t halt shipments despite patient deaths

STAT · 2d

Patient dies in Sarepta gene therapy trial, adding to safety concerns

· 1d

Sarepta tells FDA it won’t halt shipments despite patient deaths

The FDA said in a statement Friday night that officials met with Sarepta and requested it suspend all sales but “the company refused to do so.”

· 1d

Exclusive: US FDA to ask Sarepta to stop shipments of gene therapy Elevidys, source says

· 1d

Sarepta says it won't comply with FDA request to stop shipping gene therapy Elevidys

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 dystrophy over safety issues, even as patients and investors expressed growing concern about the company’s decision-making.

STAT1dOpinion

We are mothers of Duchenne patients. Recent setbacks with Sarepta must not stop progress

As mothers of children with this disease, we have wept helplessly in recent months as friends — fellow members of a club we never asked to join — said goodbye to their sons, the babies they once held in their arms, whose dreams they held in their hearts until Duchenne robbed them of working muscles or a healthy future.

Muscular Dystrophy News3d

Dosing begins in gene therapy trial for DMD-linked heart disease

The first patient has been dosed in a clinical trial testing Sardocor's one-time gene therapy for cardiomyopathy associated with Duchenne MD.