Carmen Leon Astudillo, MD

This is an open-label gene transfer therapy study evaluating the safety of and expression from delandistrogene moxeparvovec in participants with DMD. The maximum participant duration for this study is 156 weeks.

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