Positive topline data from pivotal phase III study of setmelanotide in BBS and Alstrom syndrome Dec. 23, 2020
BioCryst reports encouraging phase I data on BCX-9250 for fibrodysplasia ossificans progressiva Dec. 22, 2020
Low-dose cohort data reported from phase I/II study of AXO-AAV-GM1 for GM1 gangliosidosis Dec. 16, 2020
First phase III study of dasiglucagon in congenital hyperinsulinism does not meet primary endpoint Dec. 16, 2020
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