lysosomal storage disorders

'Clinical intuition' led FDA advisors to back Zevra's rare disease med

Zevra’s rare disease drug seems to be on the path to approval after gaining the backing of an FDA advisory committee, although the experts were split.

Annalee Armstrong 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

Resarch Center Chiesi Farmaceutici SpA Parma

Chiesi creates Boston rare disease unit ahead of Fabry launch

Feb 7, 2020 8:45am