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lysosomal storage disorders
Biotech
Azafaros' €132M series B will fund brain-penetrant drug trials
Azafaros has secured 132 million euros in series B funds that the Dutch biotech will use to push its brain disease drug through two late-stage trials.
James Waldron
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
CRISPR corrects genetic disease before and after birth in mice
Jul 13, 2021 5:00am
