Carmen Leon Astudillo, MD

Cohort 8 (non-ambulatory participants) is currently enrolling new participants. Enrollment for Cohorts 1 through 7 has been completed. This is an open-label gene transfer therapy study evaluating the safety of and expression from delandistrogene…

Investigator

Carmen Leon Astudillo

Status

Accepting Candidates

Ages

2 Years - N/A

Sexes

Male

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