Press release originally posted to the MDA website on Jan. 27, 2026 Organizations Join Forces to Map How Human Muscle Regenerates Muscular Dystrophy Association-led research collaboration with FSHD Society, LGMD2L […]
January 12, 2026, Originally posted to Business Wire Highlights: Apitegromab Biologics License Application (BLA) resubmission and U.S. launch, following FDA approval, are anticipated in 2026 for the treatment of children […]
Nov. 26, 2025 Exciting news this week: Altay Therapeutics’ DX5057 has received Orphan Drug status from the U.S. FDA. DX5057 is currently the only oral DUX4 inhibitor in development for […]
− First patient with facioscapulohumeral muscular dystrophy (FSHD) dosed with Restem-L umbilical lining modified progenitor cells (UMPCs) − The Phase 1/2a study will evaluate the safety and preliminary efficacy of […]
Author: Dr. Raymond A. Huml, Vice President, Rare Disease Strategy, Sciensus The Rare Trials Summit in Boston, held on September 9 & 10, 2025, was the inaugural event driven by […]
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