by Elleine Allapitan | Jul 5, 2023
FACTS Summary: Phase I gene therapy for Fabry...
by Elleine Allapitan | Jul 5, 2023
GTX-102 Summary: A phase 1/2 open-label, multiple-dose, dose-escalating clinical trial of the safety and tolerability of GTX-102 in pediatric patients with Angelman Syndrome...
by Elleine Allapitan | Jul 5, 2023
ST-920-201 Dose-ranging study of ST-290, an AAV2/6 Human Alpha Galactosidase – a gene therapy in subjects with Fabry disease DOSE-RANGING STUDY OF ST-920, AN AAV2/6 HUMAN ALPHA GALACTOSIDASE A GENE THERAPY IN SUBJECTS WITH FABRY...
by Elleine Allapitan | Jul 5, 2023
REN001 Summary: A double-blind, placebo-controlled, study to evaluate the efficacy and safety of 24 weeks treatment with REN001 in patients with primary mitochondrial myopathy...
by Elleine Allapitan | Jul 5, 2023
PTC923-MD-003-PKU Summary: A phase III study of PTC 923 in subjects with...
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