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Propionic Acidemia
Clinical trial pipeline · Data from ClinicalTrials.gov
See which Propionic Acidemia trials you may qualify forClinical trial pipeline · Data from ClinicalTrials.gov
See which Propionic Acidemia trials you may qualify forBackground: People s bodies need to break down food into the chemicals. These chemicals are used for energy and growth. Some people cannot process all chemical…
The main purpose of this study is to evaluate the long-term safety of mRNA-3927 administered to participants with propionic acidemia (PA) who have previously pa…
This is a non-interventional, observational, global, multicenter, study describing participant characteristics, clinical outcomes, and event rates in participan…
This 3-part, Phase 1/2 study is designed to characterize the safety, tolerability, and pharmacological activity (as assessed by biomarker measurements) and to d…
CoRDS, or the Coordination of Rare Diseases at Sanford, is based at Sanford Research in Sioux Falls, South Dakota. It provides researchers with a centralized, i…
To obtain short-term and long-term clinical safety information, in pediatric and adult patients with PA and MMA treated with Carbaglu®.
The natural history of patients with PA-AMV has not been systematically studied before and there is no published data in the literature about this condition sin…
Newborn screening (NBS) is a global initiative of systematic testing at birth to identify babies with pre-defined severe but treatable conditions. With a simple…
Early Check provides voluntary screening of newborns for a selected panel of conditions. The study has three main objectives: 1) develop and implement an approa…