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BioWorld Science, Endocrine/metabolic
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Orphazyme reports topline phase II/III data on arimoclomol in Niemann-Pick disease Type C

Sep. 28, 2018

Zealand initiates phase III trial of dasiglucagon for severe hypoglycemia in children

Sep. 28, 2018

Ultragenyx reports topline cohort 2 data from phase I/II study of DTX-301 in OTC deficiency

Sep. 28, 2018

Alnylam announces positive interim analysis results from ENVISION phase III study of givosiran

Sep. 28, 2018

Researchers develop viable murine model of cblC deficiency

Sep. 27, 2018

Newly developed ISOD mouse model mimics human disease

Sep. 27, 2018

Phase I/II clinical trial of OT-58 recruiting patients with CBSDH

Sep. 27, 2018

JCR's agalsidase beta biosimilar approved in Japan for Fabry disease

Sep. 26, 2018

Targeting hepatic OGA ameliorates hyperammonemia researchers find

Sep. 26, 2018

Researchers develop a SLC6A8 deficiency rat model

Sep. 26, 2018

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