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- Opinion: Capricor’s Duchenne muscular dystrophy drug and the countless tiny decisions that can make or break a study“Regulatory flexibility can be exercised in rare disease without sacrificing rigor,” writes Mindy Leffler, whose son has Duchenne muscular dystrophy.STAT · 51m ago↗ source
- FDA approves Regeneron’s garetosmab for ultra-rare bone-forming diseaseThe FDA has granted approval to Regeneron's antibody garetosmab, which targets activin A, for the treatment of fibrodysplasia ossificans progressiva (FOP). FOP is an ultra‑rare disorder that causes…