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- Why pediatric rare oncology outperforms in a volatile biotech marketReal progress happens where unmet medical need is greatest and patient impact can be clearly measured. Pediatric rare cancers exemplify these criteria and have therefore generated continued investm…BioSpace · 26m 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…