BOB体育

This is an open-label, single-arm, multicenter clinical study to evaluate the effectiveness and safety of risdiplam administered as an early intervention in pediatric participants with spinal muscular atrophy (SMA) and 2 SMN2 copies who have鈥�

Investigator

Carmen Leon Astudillo

Status

Accepting Candidates

Ages

3 Months - 24 Months

Sexes

All

The study will evaluate the safety and efficacy of delandistrogene moxeparvovec gene transfer therapy in non-ambulatory and ambulatory males with DMD. This is a randomized, double-blind, placebo-controlled 2-part study. Participants will be in the鈥�

Investigator

Carmen Leon Astudillo

Status

Accepting Candidates

Ages

N/A - N/A

Sexes

Male

The study will evaluate the safety and efficacy of gene transfer therapy in boys with DMD. It is a randomized, double-blind, placebo-controlled study. The participants who are randomized to the placebo arm will have an opportunity for treatment with鈥�

Investigator

Carmen Leon Astudillo

Status

Accepting Candidates

Ages

4 Years - 7 Years

Sexes

Male