Sunday, February 9, 2025

Evrysdi® (risdiplam) Shows Remarkable Progress in Pre-symptomatic SMA Infants in RAINBOWFISH Study

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Positive results from the ongoing RAINBOWFISH study, evaluating the effectiveness and safety of Evrysdi® (risdiplam) in infants with pre-symptomatic Spinal Muscular Atrophy (SMA), were presented by Roche. In this study, 80% of babies achieved a significant milestone, sitting without support for at least five seconds after a year of treatment with Evrysdi. This achievement is especially remarkable because, without treatment, these infants would never be expected to achieve this developmental milestone. Importantly, all infants in the study could swallow and feed orally, and none required permanent ventilation.

Evrysdi stands out as the sole non-invasive therapy for SMA, providing hope for improved motor function in infants. The study encompassed 26 babies with two or more copies of the SMN2 gene, a marker that indicates disease severity. It demonstrated the critical importance of early intervention, as motor neuron loss can be initiated before symptom onset in SMA patients. Evrysdi’s impact was assessed using the Bayley Scales of Infant and Toddler Development, third edition (BSID-III), with the primary efficacy population achieving the study’s primary endpoint.

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This groundbreaking treatment offers promise to infants who, without intervention, would face significant mobility challenges associated with SMA. Evrysdi has been approved in over 100 countries and has positively impacted over 11,000 patients worldwide, marking a significant milestone in the field of SMA therapy.


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