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Glycogen Storage Diseases
Clinical trial pipeline · Data from ClinicalTrials.gov
See which Glycogen Storage Diseases trials may be worth asking aboutClinical trial pipeline · Data from ClinicalTrials.gov
See which Glycogen Storage Diseases trials may be worth asking aboutNorth America
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This is a Phase 1, multicenter, open-label study to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of DNL952 in adult parti…
Pompe disease is traditionally considered a lysosomal myopathy. However, increasing experimental and clinical evidence suggests involvement of the entire motor…
The purpose of this study is to evaluate the safety, pharmacodynamics (PD), and exploratory clinical efficacy of S-606001 in adult participants with LOPD as an…
The Pompe Registry is a global, multicenter, international, longitudinal, observational, and voluntary program for patients with Pompe disease, designed to trac…
The aim of this observational study is to evaluate the impact of raw cornstarch supplementation on postprandial glycemic response in adult patients with Glycoge…
The goal of this study is to determine if the Lactate Plus meter is accurate compared to lab lactate levels, and to determine if the Accu chek guide glucometer…
Primary Objective: To describe the effect of routine practice with alglucosidase alfa in patients with IOPD ≤6 months of age, on invasive ventilation-free surv…
Empagliflozin Treatment of GSD-1b patients