CLOSED TO ENROLLMENT – DATA BEING ANALYZED
Principal Investigator: David Lynch, MD, PhD, Children’s Hospital of Philadelphia
This study involves six visits to Children’s Hospital of Philadelphia for in-clinic assessments including various performance measures, questionnaires and a neurology exam. These visits are spaced out over approximately six months. All study participants will be treated with a pulse of an oral steroid called methylprednisolone.
To enroll, patients must be able to walk 25 feet with or without an assistive device, and they must be must be over 5 years old with a genetic confirmation of Friedreich's ataxia. The purpose of this study is to see if methylprednisolone helps patients improve their performance scores, and to see if the medication is safe and tolerable in people with FRDA.
For more information about this study, please contact the program at 267-426-7584 or email us at FAProgram@email.chop.edu