NCT01703988 - An Open-label Safety, Tolerability and Dose-Range Finding Study of Multiple Doses of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy | Biotech Hunter

Enrollment

Participants

Timeline

Start Date

October 31, 2012

Primary Completion Date

January 31, 2015

Study Completion Date

January 31, 2015

Conditions

Spinal Muscular Atrophy

Interventions

DRUG

Nusinersen

Single IT injection for each dose

Trial Locations (4)

Columbia University Medical Center, New York

UT Southwestern Medical Center - Children's Medical Center Dallas, Dallas

University of Utah School of Medicine, Salt Lake City

Boston Children's Hospital, Boston