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A Study to Investigate the Pharmacokinetics and Safety of Risdiplam in Infants With Spinal Muscular Atrophy — Stella
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A Study to Investigate the Pharmacokinetics and Safety of Risdiplam in Infants With Spinal Muscular Atrophy
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A Study to Investigate the Pharmacokinetics and Safety of Risdiplam in Infants With Spinal Muscular Atrophy
Phase 2 — Testing in a larger group (up to a few hundred people) to further evaluate safety and begin to assess effectiveness.
Trial locations
(13 sites)
United States
Ann and Robert H. Lurie Children Hospital of Chicago, Chicago, Illinois
University Of Michigan, Ann Arbor, Michigan
Clinic for Special Children., Gordonville, Pennsylvania
Belgium
Hopital Universitaire des Enfants Reine Fabiola, Brussels
CHR Citadelle, Liège
Canada
Children'S Hospital of Eastern Ontario, Ottawa, Ontario
Germany
Universitatsklinikum Essen, Essen
Italy
Fondazione Serena Onlus - CENTRO CLINICO NEMO, Milano, Emilia-Romagna
Fondazione Policlinico Univeristario A. Gemelli, ROMA, Emilia-Romagna
Netherlands
UMC Utrecht, Utrecht
Norway
OUS (Oslo University Hospital), Rikshospitalet, Oslo
Poland
Uniwersyteckie Centrum Kliniczne, Gdansk
Instytut Pomnik - Centrum Zdrowia Dziecka, Warsaw