SMA THERAPIES I

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SMA type 1 (SMA1) is marked by a rapid, precipitous loss of motor neurons; thus, it is critical that a disease modifying therapy achieves an immediate pharmacological effect to arrest further motor neuron loss. In the pivotal nusinersen phase 3 study (ENDEAR), ∼10% of subjects died or required permanent ventilation by 2 months, the time required to complete four loading doses over the initial 2-month loading dose interval, and almost one-third of subjects died or required permanent ventilation by 6 months from dosing.

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