An Open Label Study of Gene Therapy Product (Vesemnogene Lantuparvovec) in Spinal Muscular Atrophy

PHASE1/PHASE2RecruitingINTERVENTIONAL

Enrollment

Participants

Timeline

Start Date

October 20, 2024

Primary Completion Date

October 30, 2027

Study Completion Date

October 30, 2027

Conditions

Spinal Muscular Atrophy

Interventions

BIOLOGICAL

vesemnogene lantuparvovec

Exploratory study evaluating the safety and efficacy of vesemnogene lantuparvovec in patients with SMA.

Trial Locations (1)

RECRUITING

Kunming Hope of Health Hospital, Kunming