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Home » Topics » BioWorld Science, Endocrine/metabolic

BioWorld Science, Endocrine/metabolic
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Preliminary data presented from phase II study of MGTA-456 in inherited metabolic disorders

Feb. 8, 2019

Sanofi Genzyme presents long-term safety data for avalglucosidase alfa in Pompe disease

Feb. 8, 2019

Interim data presented from first-in-human study of AT-GAA in patients with Pompe disease

Feb. 8, 2019

AAV9 vector encoding modified HEXB shows promise in vivo for Tay-Sachs disease

Feb. 8, 2019

Early cardiac hypertrophy seen in murine model of Pompe disease

Feb. 7, 2019

Updated clinical data presented from ongoing studies of AVR-RD-01 gene therapy for Fabry disease

Feb. 7, 2019

ArmaGen arylsulfatase A fusion protein shows promise for metachromatic leukodystrophy

Feb. 7, 2019

Denali enzyme therapy for MPS II crosses the blood-brain barrier

Feb. 7, 2019

Valerion Therapeutics presents clinical data on VAL-1221 for Pompe disease

Feb. 6, 2019

ICMR and collaborators identify new PTP-1B inhibitors

Feb. 5, 2019
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