Rare Diseases Update – September 25, 2025: Selumetinib, Del-zota, Garetosmab and More
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YouTubeLucidQuest
Rare Diseases Update – September 25, 2025: Selumetinib, Del-zota, Garetosmab and More
From the US to Australia and New Zealand, this week’s roundup spotlights pivotal regulatory moves, late-stage wins, and modality-first signals in ultra-rare diseases—spanning NF1-PN, DMD exon 44, TED, PKU/CKD, FOP, Barth syndrome, Alexander disease, and Huntington’s. 🔬 Key highlights: 🏛️ FDA expands selumetinib (KOSELUGO) to ...
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