Adrenomyeloneuropathy research hub

A Phase 1/2 Randomized, Blinded, Dose-escalation Study to Evaluate the Safety and Efficacy of Intrathecal Administration of AAV9-ABCD1 Gene Therapy (SBT101) in Adult Patients with Adrenomyeloneuropathy

This study is testing a new gene therapy called SBT101 for adult men with a condition called adrenomyeloneuropathy. Researchers want to see if it's safe, what dose works best, and if it helps with symptoms like walking problems. It's an early-stage study.