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- Pediatric rare cancer research draws steady investment despite biotech market volatilityThe article notes that pediatric rare cancers continue to attract investment because they address the greatest unmet medical needs and allow clear measurement of patient impact. Even as the biotec…
- 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…