First-of-its-Kind gene therapy trial for rare disease heart damage
NCT ID NCT06483802
Summary
This was an early-stage study to test a new gene therapy called ASP2016 for heart problems in adults with Friedreich ataxia, a rare genetic disease. The main goals were to check if the treatment was safe and how well people could tolerate it. Participants would receive a single infusion of the therapy along with steroid pills to help their body accept it, and their heart health would be monitored closely for over a year.
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