Search clinical trials for: OTQ923

Total 6 results

Novartis Pharmaceuticals

Not yet recruiting

Long-term Follow-up (LTFU) of Patients Treated With Genome-edited Autologous Hematopoietic Stem and Progenitor Cells (HSPC)

Sickle Cell Disease
Novartis Pharmaceuticals

Active, not recruiting

Study of Safety and Efficacy of Genome-edited Hematopoietic Stem and Progenitor Cells in Sickle Cell Disease (SCD)

Sickle Cell Disease

United States
PTC Therapeutics

Recruiting

A Long-Term Safety Study of PTC923 in Participants With Phenylketonuria

Phenylketonuria

United States, Germany, Spain, Australia, Brazil, United Kingdom, Denmark, Mexico, Portugal, Turkey, Canada, Georgia, Italy, Netherlands, Japan
PTC Therapeutics

Completed

A Study of PTC923 in Participants With Phenylketonuria

Phenylketonuria

Spain, United States, Australia, Germany, United Kingdom, Brazil, Denmark, Canada, France, Georgia, Italy, Mexico, Netherlands, Portugal, Turkey
PTC Therapeutics

Completed

A Study of PTC923 (CNSA-001) in Primary Tetrahydrobiopterin (BH4) Deficient Participants With Hyperphenylalaninemia

BH4 Deficiency | Hyperphenylalaninemia

United States
PTC Therapeutics

Not yet recruiting

A Study of Sepiapterin in Participants With Phenylketonuria (PKU) (EPIPHENY)

Phenylketonuria

United States

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