Muscle Owl Talks Ep81: Duchenne After 18; Avoiding Infantilisation of Condition
January 3, 2018
Muscle Owl Talks Ep65: SMA Awareness Month 2017 (August 2017)
August 22, 2017
Peter Speaks with Jo Eames at the Action Duchenne Conference
January 16, 2018
Resolaris Granted Orphan Drug Designation for LGMD & FSHMD
March 17, 2017
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.