Sarepta's Duchenne Therapy Sparked Fears
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Roche Holding AG paused all shipments of the controversial gene therapy Elevidys following a similar action by the embattled US biotechnology company that developed the treatment.
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.
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.
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.
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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 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.