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