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lysosomal storage disorders
Research
NIH gene editing fixes Tay-Sachs mutation in mice
Flipping an adenine to a guanine restored the function of a key metabolic enzyme, lowering toxic levels of built-up lipids.
Darren Incorvaia
Aug 19, 2025 4:22pm
Azafaros' €132M series B will fund brain-penetrant drug trials
May 13, 2025 7:10am
Denali rare disease drug clears safety study, reduces biomarkers
Feb 6, 2025 12:05pm
'Clinical intuition' led FDA advisors to back Zevra's rare disease med
Aug 5, 2024 10:35am
Takeda eyes cures for billion-dollar gene therapy spending spree
May 4, 2022 4:00am
Avrobio gene therapy shows signs of durability in rare disorder
Feb 9, 2022 10:46am
