Marco Meglio

The Expanded Access Program will offer treatment for patients with ALS who have completed the phase 3 clinical trial and will not interfere with data or regulatory timelines.

Results of the phase 2 study support continued dose escalation of SRP-5051 and further clinical development.

Nearly all patients in this cohort achieved a clinically meaningful >3-point increase during the study period, demonstrating a consistent response to the gene therapy.