Evrysdi Research Updates from Genentech

New 2-year data show Evrysdi continues to demonstrate improvement or maintenance of motor function in people aged 2-25 years with Type 2 or 3 SMA Genentech, a member of the Roche Group, recently shared new exploratory 2-year, longer-term data from Part 2 of SUNFISH, a global placebo-controlled study evaluating Evrysdi (risdiplam) in people aged 2-25 […]

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Zolgensma Research Updates from Novartis Gene Therapies

New Zolgensma data demonstrate age-appropriate development when used early, real-world benefit in older children, and durability 5+ years post-treatment Novartis Gene Therapies recently shared new data that reinforce the transformational benefit of Zolgensma (onasemnogene abeparvovec), a one-time treatment for spinal muscular atrophy (SMA). The overall safety profile remains favorable following pre-symptomatic treatment, in the long-term

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Cure SMA Publishes Quality of Life in Teens and Young Adults Manuscript in Orphanet Journal of Rare Diseases

Current knowledge regarding clinical meaningfulness and quality of life amongst teens and young adults with spinal muscular atrophy (SMA) is limited. Much of the available qualitative data on this population has been obtained from the perspective of the parents/caregivers rather than the individuals themselves. Knowing this, Cure SMA is pleased to announce the publication of a manuscript,

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Community Spotlight: I am…Viola Dwyer

February 28 is Rare Disease Day. Throughout the month—in recognition of our Redefining Rare initiative—we have been posting stories about SMA community members, showcasing their greatest passions, hobbies, and tidbits of their life. Viola Dwyer is from a suburb of Philadelphia and recently pivoted from her corporate job in the financial sector to creating a

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Jessica’s Story: Remembering Piper Grace

February 28 is Rare Disease Day. Throughout the month—in recognition of our Redefining Rare initiative—we will be posting stories about SMA community members, showcasing their greatest passions, hobbies, and tidbits of their life. This guest post is written by Jessica Hoefler whose daughter, Piper, passed away in May 2020, shortly after being diagnosed with SMA

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Results from Genentech’s Evrysdi Study in Infants with Type 1 SMA Published in NEJM

Genentech, a member of the Roche Group, announced in a press release that Evrysdi™ (risdiplam) data from the dose finding Part 1 of the pivotal FIREFISH study in infants with symptomatic Type 1 spinal muscular atrophy (SMA) were published in the February 24, 2021 online issue of the New England Journal of Medicine (NEJM). The data show

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Cure SMA Launches Virtual Therapy Program for Adults with SMA

Adults with spinal muscular atrophy (SMA) are invited to take advantage of Cure SMA’s latest support resource, the Adults with SMA Virtual Therapy Program, which has been generously sponsored by Genentech and the Dhont Family Foundation. The Adults with SMA Virtual Therapy Program provides adults with SMA three complementary, 30-minute live video sessions with a

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Community Spotlight: I Am…Kelsey Roberts

February 28 is Rare Disease Day. Throughout the month—in recognition of our Redefining Rare initiative — we will be posting stories about SMA community members, showcasing their greatest passions, hobbies and tidbits of their life. Kelsey Roberts is a determined, kind, and passionate 18-year-old from Massachusetts. As a teenager, she was diagnosed with spinal muscular

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