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approvalsWatch: Neurology 030117

US FDA Approvals

Nusinersen
The US FDA has approved nusinersen for the treatment of spinal muscular atrophy in children and adults. The injection is administered into the fluid surrounding the spinal cord and is the first approval for this often fatal genetic disease. The approval is based on interim analysis of 82 of 121 infants, which showed a 40% improvement in motor milestones with treatment versus 0% in controls, and supportive findings from additional uncontrolled clinical studies.