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

Ages

3 Months - 24 Months

Sexes

All

The purpose of this study is to conduct an early clinical evaluation of the Relay Branch System, which will provide initial insight into the clinical safety and function of the device. This Early Feasibility Study (EFS) will assess the safety and鈥�

Investigator

Martin R Back

Ages

18 Years - N/A

Sexes

All