Status and phase
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About
The primary objective of the study is to examine the efficacy of multiple doses of Nusinersen administered intrathecally in preventing or delaying the need for respiratory intervention or death in infants with genetically diagnosed and presymptomatic spinal muscular atrophy (SMA). Secondary objectives of this study are to examine the effects of Nusinersen in infants with genetically diagnosed and presymptomatic SMA.
Enrollment
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Inclusion and exclusion criteria
Key Inclusion Criteria:
Key Exclusion Criteria:
Note: Other protocol defined Inclusion/Exclusion criteria may apply.
Primary purpose
Allocation
Interventional model
Masking
25 participants in 1 patient group
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Data sourced from clinicaltrials.gov
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