Prothena plans phase III study of birtamimab for patients with Mayo stage IV AL amyloidosis Feb. 2, 2021
FDA clears IND for phase I/II study of VX-880 stem cell-derived therapy for type 1 diabetes Feb. 1, 2021
PRV-3279 prevents immunogenicity in a preclinical model of gene therapy for Pompe disease Jan. 29, 2021
Primary endpoint met in ACTIVATE-T trial of mitapivat in transfused patients with PK deficiency Jan. 27, 2021
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