Atossa Therapeutics Highlights (Z)-Endoxifen Potential in Duchenne Muscular Dystrophy Research
SEATTLE, May 20, 2026 Atossa Therapeutics announced that a new scientific manuscript exploring the utrophin-modulation potential of (Z)-endoxifen in...
Duchenne muscular dystrophy
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GEMMABio Expands Gene Therapy Pipeline for Rare Diseases
PHILADELPHIA, May 14, 2026 GEMMA Biotherapeutics (GEMMABio) unveiled its new Duchenne muscular dystrophy (DMD) gene therapy program and presented...
Edgewise Advances Muscle and Heart Disease Pipeline
BOULDER, Colo., May 7, 2026 Edgewise Therapeutics reported first quarter 2026 financial results while highlighting major progress across its...
Santhera Pharmaceuticals Gains EU Boost for AGAMREE Expansion
PRATTELN, Switzerland, April 27, 2026 Santhera Pharmaceuticals has achieved a significant regulatory milestone with the European Medicines Agency’s (EMA)...
SonoThera Showcases RIPPLE Gene Delivery Breakthrough for DMD
SOUTH SAN FRANCISCO, California – March 5, 2026 SonoThera announced new preclinical research demonstrating the potential of its proprietary...
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Avidity Advances RNA Therapies at MDA 2026
SAN DIEGO, Calif., February 26, 2026 Avidity Biosciences, Inc. (Nasdaq: RNA) announced that it will present one oral and...
Ifetroban Gains FDA Fast Track for DMD Heart Disease
NASHVILLE, Tenn., Feb. 4, 2026 —Cumberland Pharmaceuticals Inc. announced that the U.S. Food and Drug Administration has granted Fast...
Italfarmaco–JCR Givinostat Deal Expands Rare Disease Pharma in Japan
MILAN, Italy & HYOGO, Japan | December 24, 2025 — Italfarmaco and JCR Pharmaceuticals have announced a landmark commercialisation...
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