ENDEAVOR
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
BN44621 Hinalea 2
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
Respiratory training in Friedreich's ataxia
- Investigator
- Carmen Leon Astudillo
- Status
- Accepting Candidates