Decision day for Sarepta’s second Duchenne drug
The FDA is expected to make a decision today on Vyondys 53, a new drug aimed at treating Duchenne muscular dystrophy from Sarepta Therapeutics. If approved, it would expand the population of Duchenne patients eligible for treatment; approval would also come three years after the FDA gave a controversial green light to Sarepta's first Duchenne drug, Exondys 51.
Vyondys 53 targets an aberration in the gene sequence known as exon 53. In clinical trials, the drug, administered via a weekly infusion, was shown to increase an important muscle protein called dystrophin that is normally missing in children with Duchenne.
Vyondys 53 targets an aberration in the gene sequence known as exon 53. In clinical trials, the drug, administered via a weekly infusion, was shown to increase an important muscle protein called dystrophin that is normally missing in children with Duchenne.
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