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...
by Elleine Allapitan | Jul 5, 2023
PALOMINO Summary: A global multicenter study to assess maternal, fetal and infant outcomes of exposure to Palynziq® (Pegvaliase) during pregnancy and...
by Elleine Allapitan | Jul 5, 2023
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...
by Elleine Allapitan | Jul 5, 2023
G.R.I.T. STudy Getting global Rare disease Insights through Technology Summary: The G.R.I.T. Study is the first global app-based registered clinical trial for patients with metabolic disorders. It uses a mobile health journal, Zamplo, to record patient-reported...
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