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.

Shares of Sarepta Therapeutics (NASDAQ: SRPT) plunged 26.85% in Friday pre-market after a clinical trial participant died following treatment with a gene therapy, marking the third death linked to the company's experimental treatments.

Sarepta Therapeutics to lay off 493 workers, including 80 in Ohio, amid FDA probe, stock plunge, and concerns over Duchenne gene therapy Elevidys.

The FDA will request Sarepta Therapeutics stop all shipments of Elevidys, its gene therapy for Duchenne muscular dystrophy, following two deaths tied to the product.

New research has identified the enzyme glutamate dehydrogenase 1 (GLUD1) as a new therapeutic target for Duchenne muscular dystrophy (DMD). In preclinical DMD mouse models, investigators demonstrated that inhibiting GLUD1 significantly enhances muscle strength and coordination,

Three patients with a muscle-wasting disease died from liver failure after taking the therapy, Ele­vidys, or a similar treatment.

A company spokesperson told Bloomberg that a 51-year-old patient died of acute liver failure last month in an early-stage trial of a gene therapy to treat limb-girdle muscular dystrophy.

Sarepta Therapeutics appears to have right-sized itself after laying off over a third of its staff, announcing a significant pipeline shift, and adding a black box warning to its Duchenne muscular dystrophy gene therapy Elevidys.