FDA grants fast-track designation for Sarepta's muscular dystrophy treatment
By Alexander Bueso
Date: Friday 24 Jul 2020
(Sharecast News) -
Sarepta Therapeutics's investigational gene therapy treatment SRP-9001 for Duchenne Muscular Dystrophy has received fast-track designation from America's Food and Drug Administration.
The therapy is designed to deliver the missing mycro-dystrophin encoding gene needed to produce the protein responsible for maintaining muscle tone.
Safety and tolerability data from a four patient trial at the one-year stage was recently reported on in JAMA Neurology.
Another study, which was randomized, double-blind, and placebo-controlled was ongoing with the results expected for early 2021.
Following a brief move higher, as of 1420 BST shares of Sarepta were drifting lower by 0.5% to $160.86.
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