Open-label Pilot Study of Methylprednisolone for the Treatment of Patients With Friedreich Ataxia (FRDA)

Open-label Pilot Study of Methylprednisolone for the Treatment of Patients With Friedreich Ataxia (FRDA). Sponsor: Children's Hospital of Philadelphia, Collaborator: Friedreich's Ataxia Research Alliance. First received: April 16, 2015

This study will explore whether methylprednisolone treatment is safe, well-tolerated, and beneficial in patients that are diagnosed with Friedreich Ataxia (FRDA). The study will also explore if methylprednisolone has any effects on biomarkers associated with FRDA. All subjects in the study will receive the same steroid treatment.
This study will take place at the Children's Hospital of Philadelphia as an outpatient trial in 5 children who are at least 5 years and less than 10 years of age, and in 5 adults ages 45 years and older, with genetically confirmed FRDA.

German regulator puts UniQure gene therapy appraisal on hold

German regulator puts UniQure gene therapy appraisal on hold. REUTERS. FRANKFURT, April 17

(Reuters) - A German regulator for new drugs has suspended the assessment of the Western world's first gene therapy, UniQure's Glybera, after an adviser to the European drugs watchdog voiced concern over the treatment. (Read more)