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Sarepta Tells Patients To Stand Down, Golodirsen Future 'Our Responsibility'

 
• By Derrick Gingery

Patient advocates asked what they could do after US FDA’s complete response letter for the proposed Duchenne drug, but Sarepta's CEO said the company would get the product back on track to approval itself, a marked contrast to engaged role the community played the last time the firm ran into a roadblock at the agency.

MUSCULAR DYSTROPHY words written on label tag with medicine,syringe,keyboard and stethoscope
Sarepta's CEO also said he believed no advisory committee was held for golodirsen because the FDA was moving toward approval. • Source: Shutterstock

Sarepta Therapeutics Inc. wants the Duchenne muscular dystrophy community to sit and wait while the company determines its next steps for golodirsen, a position where the group is not comfortable.

When asked what DMD patients, parents and advocates could do following the US Food and Drug Administration's surprising complete response letter for its latest therapy, Sarepta president and CEO Douglas Ingram told them it

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