Pulmonary Arterial Hypertension Clinical Trials

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AAV Gene Therapy Study for Subjects With PKU

This is a Phase 1/2, open-label, dose escalation study to evaluate the safety, efficacy and tolerability of BMN 307 in adult PKU subjects with PAH deficiency. Participants will receive a single administration of BMN 307 and will be followed for safety and efficacy.

Heeba Test Trial  

PKU is an inherited disorder caused by abnormalities in the gene coding for the phenylalaninehydroxylase (PAH) enzyme, which increases the levels of Phenylalanine (Phe), a building blockof proteins in the blood. If PKU is not adequately treated, Phe can increase to harmful levels inthe body, causing injury to the brain …

Sujana-A Phase 1/Phase 2 open-label study to evaluate the safety, tolerability, and efficacy of a single intravenous administration of an investigational medication in adult participants with phenylketonuria (PKU)  

PKU is an inherited disorder caused by abnormalities in the gene coding for the phenylalaninehydroxylase (PAH) enzyme, which increases the levels of Phenylalanine (Phe), a building blockof proteins in the blood. If PKU is not adequately treated, Phe can increase to harmful levels inthe body, causing injury to the brain …

Pharmacokinetics Pharmacodynamics and Safety Profile of Understudied Drugs Administered to Children Per Standard of Care (POPS)

The study investigators are interested in learning more about how drugs, that are given to children by their health care provider, act in the bodies of children and young adults in hopes to find the most safe and effective dose for children. The primary objective of this study is to …