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Neurological Measures of Progression in Children with FA

Children's Hospital of Philadelphia, University of Florida and University of California Los Angeles are seeking children with FA for a 3 yr study to help advance our understanding of disease progression and validate outcome measures that can be used in future clinical trials.

This study is funded by the Food and Drug Administration (FDA) and the Friedreich’s Ataxia Research Alliance (FARA).

It is a natural history study with the following objectives:

Collect data twice a year to understand progression of FA and be able to quantify change in progression over time. Develop and validate outcome measures (timed walk tests, vision tests) that can be used in future clinical trials Make design and implementation of clinical trials more efficient and effective Collect biological samples such as cheek swabs and blood to identify and study biomarkers involved with the disease process and quantify the change in these markers over time Analyze disease progression in FA affected tissues using innovative and noninvasive measures

Key Inclusion Criteria:
You/your child have a confirmed diagnosis of Friedreich’s ataxia
You/your child are between the ages of 2 and 18 years of age

To learn more about the Inclusion and Exclusion Criteria and the process for participation in the study, please contact the Clinical Research Coordinator with any questions.

Children's Hospital of Philadelphia, PA - View the CHOP Study Flyer
Contact: Cassandra Strawser, 215-590-2314 -

University of California Los Angeles, CA
Contact: Aaron Fisher, 310-206-8153 -
or Arjun Sarkar -

University of Florida, Gainesville, FL
Contact: Samantha Norman, 352-273-8218 -  

For more information visit -

Friedreich Ataxia - Clinical Outcome Measures

Description: This is a multicenter natural history study to provide a framework for facilitating therapeutic interventions; including the development of valid yet sensitive clinical measures crucial to outcome assessment of patients with Friedreich's Ataxia and supporting genetic modifier studies, biomarker studies, frataxin protein level assessments and a sample repository.

Key Criteria: Ages 4-80 years

Location: Multiple sites (US, Australia and Canada) in the Collaborative Clinical Research Network -


Mitochondrial Disease Participants Needed for New Research Study at CHOP and HUP

Description: This is a study to investigate the relationship between mitochondrials disease, obesity and risk of diabetes using non-invasive MRI methods and blood glucose tests.

Key Criteria: Ages 18-65 years

Location: Children's Hospital of Philadelphia and University of Pennsylvania, Philadelphia, PA -

Please review the study flyer below, and contact the Study Coordinator with any questions.

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Biomarkers in FA

Description: This is a 5-year observational study that will measure heart function, exercise tolerance, speech, swallowing, and motor function.

Key Criteria: Ages 8-70 years

Location: University of Florida, Gainesville, FL -

Please review the study flyer below, and contact the Study Coordinator with any questions.

Can't see this document? Click Here


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