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- STAT+: FDA approves a new gene therapy for Sanfilippo syndrome, an ultra-rare diseaseThe FDA approved a new gene therapy for Sanfilippo syndrome type A, an ultra-rare disease sometimes known as childhood Alzheimer’s.STAT · 1mo ago↗ source
- Novo signs $1.4 billion deal with Orbis Medicines to develop oral macrocycle cardiometabolic drugsNovo, which recently rebranded from Novo Nordisk, announced a partnership with Copenhagen‑based Orbis Medicines. The agreement could provide up to $1.4 billion in upfront payments and milestone pay…