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- FDA approves Ultragenyx's gene therapy Genglycos for glycogen storage disorderUltragenyx has secured its first approved gene therapy, with the FDA clearing its asset for an ultra-rare disease that can cause life-threatening blood sugar crashes. The FDA on Wednesday granted a…Endpoints · 21m 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…