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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
- Stanford researchers create mice with half human brain tissue using cerebral organoidsScientists at Stanford have implanted human cerebral organoids into mice whose own brain regions were genetically removed, producing neuro-chimeric animals whose brains are about half human by volu…
- Skyhawk reports functional gains in Huntington's disease trial of RNA-splicing pill