FIREFISH is a 2-part, open-label clinical study in infants with Type 1 spinal muscular atrophy (SMA). It included 62 infants 2 to 7 months old.
BSID-III stands for Bayley Scales of Infant and Toddler Develoment-Third Edition.
The Bayley Scales of Infant and Toddler Development–Third Edition (BSID-III) is a gross motor scale that assesses a range of physical abilities such as sitting, rolling, and crawling.
“Evrysdi has been the right decision for our family. Kayden has just had a wonderful response.”
81% of infants (17/21) were alive and able to breathe without permanent support*
81% of infants (17/21) were alive and able to breathe without permanent support*
*Permanent support was defined as having a tracheostomy (a surgery where a tube is inserted in the front of the throat into the windpipe) or more than 21 days of either noninvasive ventilation support (16 or more hours a day) or being intubated (a procedure where a breathing tube is inserted down the throat and into the windpipe) to help with breathing, in the absence of an acute reversible event.
*Two of these infants could eat some food by mouth, but also used a feeding tube.
†One infant could not swallow at the start of the study.
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Información de prescripción de Evrysdi® (risdiplam). Genentech, Inc.
Información de prescripción de Evrysdi® (risdiplam). Genentech, Inc.
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