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Resolaris Granted Orphan Drug Designation for LGMD & FSHMD

The European Medicines Agency has granted Orphan Drug Designation to Resolaris (ATYR1940) for the treatment of all types of limb girdle muscular dystrophy (LGMD) & Facioscapulohumeral MD (FSHMD).

In muscular dystrophies like LGMD, inflammation contributes to muscle damage. The aim of the protein Resolaris is to reduce inflammation by altering the body’s immune response.

aTyr Pharma recently reported its phase Ib/II trial for Resolaris, testing it in patients with LGMD 2B, which found it to be safe and did not suppress circulating immune cells. It also displayed effectiveness, improving muscle function in 7 of the 9 treated patients after 14 weeks.

While the overall muscle strength improvement in the trial was reasonably small (6% compared to baseline/scores recorded prior to the treatment), the trial has now been extended to monitor the drug’s effect longer term.

#Science #LGMD