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sEARCH RESULTS

GTX-102

GTX-102Summary: 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 (AS)

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ST-920-201

ST-920-201Dose-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 DISEASE

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REN001

REN001Summary: A double-blind, placebo-controlled, study to evaluate the efficacy and safety of 24 weeks treatment with REN001 in patients with primary mitochondrial myopathy (PMM).

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CMVIBE TRIAL

CMVIBE TRIAL Summary: A phase 1/2A open-label dose-ranging and observer-blind placebo-controlled, safety and immunogenicity study of mRNA-1647 Cytomegalovirus vaccine in female and male participants 9 to 15 years of age and participants 16 to 25 years of age.

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AVRO-RD-02-LTF01

AVRO-RD-02-LTF01 A Long-Term Follow-up Study of Subjects With Gaucher Disease Who Previously Received AVR-RD-02 Summary: A follow-up study of subjects with Type I Gaucher Disease who previously received AVR-RD-02 gene therapy      

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