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Amino Acid Metabolism, Inborn Errors
Clinical trial pipeline · Data from ClinicalTrials.gov
See which Amino Acid Metabolism, Inborn Errors trials may be worth asking aboutClinical trial pipeline · Data from ClinicalTrials.gov
See which Amino Acid Metabolism, Inborn Errors trials may be worth asking aboutNorth America
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The primary objective of this clinical trial is to evaluate the safety and tolerability of single and multiple administrations of PL54 in patients aged 18-55 ye…
The purpose of this study is to gain a better understanding of alkaptonuria and collect medical data on patients who may later participate in new drug trials fo…
Hermansky-Pudlak Syndrome (HPS) is an inherited disease which results in decreased pigmentation (oculocutaneous albinism), bleeding problems due to a platelet a…
The aims of this collaborative, interdisciplinary research project are to understand and describe the psychological impact of the announcement of a rare, seriou…
Propionic acidemia is a genetic metabolic disorder characterized by metabolic acidosis, ketosis, vomiting, lethargy, cognitive impairment, and risk of death. It…
The primary purpose of this study is to assess the safety and tolerability of AG-181 in subjects with Phenylketonuria (PKU).
This is a single-site Phase 1/2 open-label umbrella clinical trial designed to evaluate the safety, tolerability, and efficacy of a single intravenous dose of L…
Phenylalanine (Phe) free protein substitutes are typically presented in ready to drink liquid or powder format and are made up with water to a set volume. Despi…