The aim of the proposed project is to evaluate whether the metabolome of patients with spinal muscular atrophy (SMA) before the initiation of treatment with nusinersen differs from the metabolome of healthy individuals and whether it changes 14 months after treatment with nusinersen.
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Metabolomic difference from healthy children
Timeframe: Beginning of study
Metabolomic change before and after treatment
Timeframe: At least 14 months of treatment