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lysosomal storage disorders
Biotech
Takeda eyes cures for billion-dollar gene therapy spending spree
Madhu Natarajan, head of Takeda’s Rare Disease Drug Discovery unit, knows that to many, cure is a “four-letter word.” But that's no reason not to try.
Annalee Armstrong
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