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BioMarin Pharmaceutical & Sanguine Biosciences -Biomarker Research Study

Description: BioMarin Pharmaceutical and Sanguine Biosciences are seeking additional individuals with FA and FA Carriers (biological parents and genetically confirmed siblings) who live in Northern California, in the extended San Francisco Bay Area, to participate in their Biomarker Research study.

The purpose of the study is to explore potential biomarkers that may serve as biochemical measurements of the effects of FA, and may be used to monitor therapeutic effects of a future treatment.

Key Inclusion Criteria: Adults 18 years and older who were diagnosed with FA prior to age 20, or a related Carrier has one FA disease allele (biological parent or genetically confirmed siblings). Individuals or Carriers who have GAA repeat length >100 and/or a single nucleotide point mutation. Patients must be able to donate blood 4 times over 4-6 months, at home visits, and must live in the extended San Francisco Bay Area. Carriers may donate blood 1 time.

To learn more about the Inclusion and Exclusion Criteria and the process for participation in the study, including the link to the Study Landing Page, see the attached flyer below.

Please contact the Clinical Research Coordinator with any questions.

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