A Study to Assess the Safety, Tolerability, Efficacy, Pharmacokinetics, and Immunogenicity of Intravenous Administration of ARGX-119 in Pediatric Participants Aged 5 to Less Than 18 Years With Spinal Muscular Atrophy
Conditions
Spinal Muscular Atrophy (SMA)
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Locations
Arkansas Children's Hospital, Little Rock, Arkansas, United States
Contact: Principal Investigator (Kapil Arya, Dr.)Rady Childrens Hospital, San Diego, California, United States
Contact: Principal Investigator (Chamindra Laverty, Dr.)Stanford University Medical Center, Stanford, California, United States
Contact: Principal Investigator (Carolina Tesi Rocha, Dr.)Connecticut Children's Medical Center, Hartford, Connecticut, United States
Contact: Principal Investigator (Gyula Acsadi, Dr.)Rare Disease Research FL LLC, Kissimmee, Florida, United States
Contact: Principal Investigator (Renata Shih, Dr.)Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois, United States
Contact: Principal Investigator (Nancy Kuntz, Dr.)University of Iowa Stead Family Children's Hospital, Iowa City, Iowa, United States
Contact: Principal Investigator (Katherine Mathews, Dr.)The Johns Hopkins Hospital, Baltimore, Maryland, United States
Contact: Principal Investigator (Jessica Nance, Dr.)Boston Children's Hospital, Boston, Massachusetts, United States
Contact: Principal Investigator (Basil Darras, Dr.)The Curators of the University of Missouri on behalf of University of Missouri Health Care, Columbia, Missouri, United States
Contact: Study coordinator (Heather McHatton) · heathermchatton@health.missouri.edu · (573) 882-7619Contact: Principal Investigator (William Arnold, Dr.)Columbia University Herbert Irving Comprehensive Cancer Center, New York, New York, United States
Contact: Principal Investigator (Claudia Chiriboga, Dr.)Rare Disease Research NC, LLC, Hillsborough, North Carolina, United States
Contact: Principal Investigator (Edward Smith, Dr.)Children's Hospital Philadelphia - Neurology, Philadelphia, Pennsylvania, United States
Contact: Principal Investigator (John Brandsema, Dr.)St. Jude Children's Research Hospital, Memphis, Tennessee, United States
Contact: Principal Investigator (Richard Finkel, Dr.)Neurology Rare Disease Center, Flower Mound, Texas, United States
Contact: Principal Investigator (Diana Castro, Dr.)Texas Children's Hospital, Houston, Texas, United States
Contact: Principal Investigator (Timothy Lotze, Dr.)Childrens Hospital of The Kings Daughters, Norfolk, Virginia, United States
Contact: Principal Investigator (Crystal Proud, Dr.)