SMA is a genetic condition that causes muscles throughout the body to break down.
This happens when the body can’t produce enough of a key protein called “survival motor neuron,” or SMN, which is needed for specialized nerves called motor neurons to function properly.
SMA is a genetic condition that causes muscles throughout the body to break down.
This happens when the body can’t produce enough of a key protein called “survival motor neuron,” or SMN, which is needed for specialized nerves called motor neurons to function properly.
Within 4 weeks of treatment with Evrysdi, SMN protein levels in the blood more than doubled, and they were maintained for Types 1, 2, and 3 SMA throughout 2 years of the pivotal studies*
When studied in animals, this protein was distributed throughout the body.
*No data available in presymptomatic SMA (under 2 months).
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Información de prescripción de Evrysdi® (risdiplam). Genentech, Inc.
Información de prescripción de Evrysdi® (risdiplam). Genentech, Inc.
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