What are you looking for?

Patient Recruitment Case Study: Phenylketonuria

A Phase 1/2 clinical trial evaluating an investigational gene therapy for adults with phenylketonuria (PKU)

THE CLINICAL TRIAL

PKU, or phenylalanine hydroxylase (PAH) deficiency, is a rare genetic disorder affecting approximately 70,000 diagnosed patients in the regions of the world where BioMarin operates and is caused by a deficiency of the enzyme PAH. This enzyme is required for the metabolism of Phe, an essential amino acid found in most protein-containing foods. If the active enzyme is not present in sufficient quantities, Phe accumulates to abnormally high levels in the blood and becomes toxic to the brain, resulting in a variety of complications including severe intellectual disability, seizures, tremors, behavioral problems and psychiatric symptoms.

The Phearless Study was a phase 1/2 open-Label, dose escalation study to deter-mine the safety and efficacy of BMN 307, an adeno-associated virus vector-mediated gene transfer of human phenylalanine hydroxylase in subjects with phenylketonuria.

Patient recruitment phenylketonuria clinical study patient brochure branding and marketing materials
Patient recruitment phenylketonuria clinical study diet guide branding and marketing materials
Patient recruitment phenylketonuria clinical study patient contact card branding and marketing materials
Patient recruitment phenylketonuria clinical study patient journey branding and marketing materials
Patient recruitment phenylketonuria clinical study gene therapy guide branding and marketing materials

Put our solutions to work for your next clinical trial.

Back Top