Sarepta Therapeutics (NASDAQ:SRPT) faces an “arduous and treacherous path” to bring its gene therapy Elevidys back to market, according to a STAT report citing a senior FDA official, suggesting the treatment’s license could be revoked amid ongoing safety concerns.

Roche Holding AG temporarily halted shipments of its muscular disorder gene therapy, Elevidys, outside the U.S., mirroring its U.S. partner Sarepta Therapeutics. The pause affects orders relying on FDA approval and applies universally to Duchenne muscular dystrophy patients,

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.

A lemonade stand is a fairly common sight for a hot summer day in July. Outside of Kitchen Concepts in Lexington, a white and yellow stand is doing more than just satisfying thirst.

Duchenne Muscular Dystrophy affects 12,000 to 15,000 children and young adults in the United States and about 300,000 worldwide. It's caused by a mutation in the dystrophin gene, which makes a ...

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.