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Classical Phenylketonuria(PKU)
Clinical trial pipeline · Data from ClinicalTrials.gov
See which Classical Phenylketonuria(PKU) trials may be worth asking aboutClinical trial pipeline · Data from ClinicalTrials.gov
See which Classical Phenylketonuria(PKU) 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 aims of this collaborative, interdisciplinary research project are to understand and describe the psychological impact of the announcement of a rare, seriou…
The primary purpose of this study is to assess the safety and tolerability of AG-181 in subjects with Phenylketonuria (PKU).
The main purpose of this trial is to evaluate the long-term efficacy of sepiapterin on preserving neurocognitive functioning in children with PKU when treatment…
Study 165-401 is a Phase 4, open-label study designed to examine the concomitant use of methotrexate (MTX) to suppress immune responses to Palynziq and improve…
The purpose of this project is to study the effectiveness of teaching teens and young women with Phenylketonuria (PKU) or Maple Syrup Urine Disease (MSUD) about…
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…
The main purpose of this study is to evaluate the long-term safety of PTC923 in participants with phenylketonuria, and to evaluate the changes from baseline in…