Congenital Disorders of Glycosylation
Clinical trial pipeline · Data from ClinicalTrials.gov
See which Congenital Disorders of Glycosylation trials may be worth asking aboutClinical trial pipeline · Data from ClinicalTrials.gov
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The purpose of this research is to study the natural history of congenital disorders of glycosylation and its causes and treatments.
Background: \- Proteins, fats, and other molecules are the body s building blocks. Many of these molecules must have sugars, or chains of sugars, attached to w…
The primary objective of this study is to evaluate the safety and tolerability of the dietary supplement, nicotinamide mononucleotide (NMN), in individuals with…
The Congenital Muscle Disease Patient and Proxy Reported Outcome Study (CMDPROS) is a longitudinal 10 year study to identify and trend care parameters, adverse…
The goal of this clinical trial is to provide continued access to GLM101 to treat PMM2-CDG in people who have previously received GLM101 in other trials and lea…
Clinical and Basic Investigations into Phosphomannomutase deficiency (PMM2-CDG) This is a natural history (observational) protocol designed to collect clinical…