Avidity Biosciences’ Treatment for Rare Form of Muscular Dystrophy Deemed Orphan Drug

February 20, 2023

Avidity Biosciences’ treatment for a rare muscular dystrophy called facioscapulohumeral muscular dystrophy (FSHD) has been given orphan drug status by the US Food and Drug Administration (FDA). The drug, if approved, would be the first for the disease, which results in deterioration of muscle function beginning in the upper extremities.

According to Emily Kimber, “A murine version of the candidate has already been shown in preclinical studies to prevent the development of muscle weakness. Now, AOC 1020 is being evaluated in the phase 1/2 FORTITUDE clinical trial in adults with FSHD, with the company expecting to share preliminary data from around half of the trial’s participants in the first half of 2024.”

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(Source: PM Live, February 15th, 2023)

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