Participants enrolled across all studies included a range of attributes:
EFFICACY is being studied in people aged 16 days to 25 years
SAFETY is being studied in people aged 16 days to 60 years
*The efficacy and safety of Evrysdi were established in 3 main studies. SUNFISH is a 2-part, placebo-controlled study in 231 adults and children aged 2 to 25 years with Type 2 or 3 SMA. FIREFISH is a 2-part, open-label study in 62 infants aged 2 to 7 months with Type 1 SMA. RAINBOWFISH is an ongoing, open-label study in 26 newborns younger than 6 weeks (at first dose). These newborns were genetically diagnosed with SMA and had not yet shown symptoms (presymptomatic SMA). A fourth study, JEWELFISH, is an open-label safety study in 174 people aged 1 to 60 years with Type 1, 2, or 3 SMA that was previously treated with approved or investigational SMA medications.
†In SUNFISH Part 1, 7 people were able to walk; in JEWELFISH, 16.
PART 1
PART 2
*Measured by the Motor Function Measure-32 Items (MFM-32) scale.
†Adults and children not taking Evrysdi took a placebo, a substance that has no active medication and is often used in studies.
‡Measured by the Revised Upper Limb Function scale.
who received the recommended dose of Evrysdi in Parts 1 and 2 were included in a pooled analysis that evaluated the effectiveness of Evrysdi
Explored the recommended dose of Evrysdi in 21 infants (aged 3 to 7 months)
Measured the safety and effectiveness of Evrysdi in 41 infants (aged 2 to 7 months) at the recommended dose
*Measured by Item 22 of the Bayley Scales of Infant and Toddler Development–Third Edition (BSID-III) gross motor scale.
†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.
had enrolled in the study
had received Evrysdi for at least 1 year and were included in the measurement of effectiveness
*Measured by Item 22 of the Bayley Scales of Infant and Toddler Development–Third Edition (BSID-III) among at least 5 infants with 2 copies of the SMN2 gene and compound muscle action potential (CMAP) amplitude ≥1.5 mV at the start of the study. CMAP is a measure of muscle activity after the nerve that controls the muscle is stimulated
*All but 3 patients enrolled in JEWELFISH received previous treatment; these 3 patients were previously enrolled in a different trial but received placebo only.
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Información de prescripción de Evrysdi® (risdiplam). Genentech, Inc.
Información de prescripción de Evrysdi® (risdiplam). Genentech, Inc.
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