A Study Evaluating the Effectiveness and Safety of Risdiplam Administered in Pediatric Patients With Spinal Muscular Atrophy Who Experienced a Plateau or Decline in Function After Gene Therapy — Stella
A Study Evaluating the Effectiveness and Safety of Risdiplam Administered in Pediatric Patients With Spinal Muscular Atrophy Who Experienced a Plateau or Decline in Function After Gene Therapy
Phase 4 — Post-approval study. The treatment has been approved, but researchers continue monitoring it in a large population over time.
Trial locations(19 sites)
United States
University of Arkansas for Medical Sciences, Little Rock, Arkansas
Valley Children's Hospital, Madera, California
Stanford Univ Medical Center, Palo Alto, California
Children's Hospital of Colorado, Aurora, Colorado
University of Florida Pediatrics, Gainesville, Florida
Children's Healthcare of Atlanta Center for Advanced Pediatrics, Atlanta, Georgia
Helen DeVos Children's Hospital at Spectrum Health, Grand Rapids, Michigan
Children'S Hospital of Philadelphia, Philadelphia, Pennsylvania
University of Texas Southwestern Medical Center, Dallas, Texas
Children's Hospital of the King's Daughter, Norfolk, Virginia
Germany
Charité - Universitätsmedizin Berlin SPZ Abteilung Neuropaediatrie, Berlin
UKGM Standort Gießen, Giessen
Israel
Soroka Medical Center, Beersheba
Schneider Children's Medical Center of Israel, Petah Tikva
Sourasky MC, Dana-Dwek Children's Hospital, Tel Aviv