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Clincial Study Patient Recruitment and Site Engagement

Phenylketonuria

Stark Raving Branding and Digital Marketing

Examining an Invetigational Gene Therapy for People Living with Phenylketonuria (PKU)

SponsorBioMarin Pharma

 

ClientPRA Health Sciences

 

IndicationPhenylketonuria

 

MaterialsStudy Branding

 Patient Education

 Patient Education

 Site Staff Materials

 Referring Physician

 Patient Retention






PKU, or phenylalanine hydroxylase (PAH) deficiency, is a 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 determine the safety and efficacy of BMN 307, an adeno-associated virus vector-mediated gene transfer of human phenylalanine hydroxylase in subjects with phenylketonuria.








Stark Raving Branding and Digital Marketing

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