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lysosomal storage disorders
Biotech
Denali rare disease drug clears safety study, reduces biomarkers
Denali's drug met its primary safety endpoints and reduced key biomarkers of Hunter syndrome in a phase 1/2 trial, the biotech announced.
Darren Incorvaia
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
CRISPR corrects genetic disease before and after birth in mice
Jul 13, 2021 5:00am
Chiesi creates Boston rare disease unit ahead of Fabry launch
Feb 7, 2020 8:45am
