An Open-label Safety and Tolerability Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy Who Previously Participated in ISIS 396443-CS1 (NCT01494701)

PHASE1CompletedINTERVENTIONAL

Enrollment

Participants

Timeline

Start Date

January 31, 2013

Primary Completion Date

February 28, 2014

Study Completion Date

February 28, 2014

Conditions

Spinal Muscular Atrophy

Interventions

DRUG

nusinersen

Administered by intrathecal (IT) injection

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