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-004-PKU Summary: A phase III study of PTC 923 in subjects with...
by Elleine Allapitan | Jul 5, 2023
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 ...
by Elleine Allapitan | Jul 4, 2023
MIGALASTAT SWITCH Summary: Evaluation of Biomarkers for patients receiving migalastat after switching from...
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