the most common disease-causing mutation that results in the most severe form of glycogen storage disease type 1a (GSD1a). GSD1a is an autosomal recessive disorder caused by mutations involved in ...
1). Preclinical programs include wholly owned mRNA therapeutics for cystic fibrosis and influenza, along with partnered programs in glycogen storage disease type 3, hepatitis B, and non-alcoholic ...
More Than 40 Adult Sickle Cell Disease Patients Now Enrolled in BEACON Trial of BEAM-101; Beam Expects to Dose 30 Patients and Present Updated ...
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