Carmen Leon Astudillo, MD

This is an open-label, single-arm, multicenter clinical study to evaluate the effectiveness and safety of risdiplam administered in pediatric participants with SMA and 2 SMN2 copies who previously received onasemnogene abeparvovec and experience a…

Investigator

Carmen Leon Astudillo

Status

Accepting Candidates

Ages

3 Months - 24 Months

Sexes

All

Investigator

Carmen Leon Astudillo

Status

Accepting Candidates

The purpose of this study is to provide a single clinical study with a uniform approach to monitoring long-term safety and efficacy in participants who received delandistrogene moxeparvovec in a previous clinical study. No study drug will be…

Investigator

Carmen Leon Astudillo

Status

Accepting Candidates

Ages

N/A - N/A

Sexes

Male