Propionic Acidemia Clinical Trial
Official title:
A Longitudinal, Exploratory, Natural History Study to Further Characterize and Describe the Signs and Symptoms of Patients With Organic Acidemias
Longitudinal, exploratory, natural history study of patients with MMA due to mut deficiency and PA to characterize the changes in blood disease biomarkers over time and the frequency and severity of clinical events related to their disease.
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Status | Clinical Trial | Phase | |
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Enrolling by invitation |
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Phase 2 | |
Terminated |
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Natural History Study of Patients With Methylmalonic Acidemia and Propionic Acidemia
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Completed |
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Recruiting |
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