Nusinersen in adults with spinal muscular atrophy

Takeaway

  • Nusinersen is associated with improvements in motor and pulmonary function in adults with spinal muscular atrophy (SMA).

Why this matters

  • Individuals with SMA lack functional copies of SMN1, resulting in a deficiency of survival motor neuron (SMN) protein; SMN2 generates SMN protein less efficiently than SM1.

  • Nusinersen, an antisense oligonucleotide that modifies SMN2 RNA splicing and increases protein production, improved motor function and survival in childhood-onset SMA; however, it has not been trialed in adults.

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