Daily medicine aims to stop devastating muscle disease in babies before it starts
NCT ID NCT03779334
Summary
This study is testing a daily oral medicine called risdiplam in infants who have been diagnosed with spinal muscular atrophy (SMA) but do not yet show symptoms. The goal is to see if starting treatment very early can help control the disease and allow babies to reach important motor milestones like sitting and standing. Infants receive the medicine daily for at least 5 years to assess its long-term safety and effectiveness.
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Contacts and locations
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Locations
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Chr de La Citadelle
Liège, 4000, Belgium
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Hospital das Clinicas - FMUSP_X
São Paulo, São Paulo, 05403-000, Brazil
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Kaohsiung Medical University Chung-Ho Hospital
Kaohsiung City, 807, Taiwan
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Nemours Children's Hospital
Orlando, Florida, 32837, United States
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Russian Children Neuromuscular Center of Veltischev
Moscow, Moscow Oblast, 125412, Russia
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Sydney Children's Hospital
Randwick, New South Wales, 2031, Australia
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Szpital Gdanskiego Uniwersytetu Medycznego
Gda?sk, 80-952, Poland
Conditions
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