Cure SMA Working Group Revises Recommendations for the Treatment of Infants Diagnosed with SMA via Newborn Screening Who Have 4 Copies of SMN2

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To help clinicians and the families they serve in the decision of when to administer therapy to infants identified with SMA via newborn screening, Cure SMA convened a working group comprised of 15 SMA experts to develop treatment guidelines. These guidelines, “Treatment Algorithm for Infants Diagnosed with Spinal Muscular Atrophy through Newborn Screening,” were originally […]

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Cure SMA Launches “Spotlight on SMA” Partnership with Neurology Reviews

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Cure SMA is pleased to announce the release of “Spotlight on SMA: The Urgent Need for Early Diagnosis in Spinal Muscular Atrophy,” a supplement developed in collaboration with Neurology Reviews. The goal of this partnership is to enhance current awareness and understanding of SMA diagnostic requirements, while emphasizing the importance of early treatment as it

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Celebrate Rare Disease Day 2020 with Cure SMA

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This year, Rare Disease Day occurs on February 29—the rarest day of all! Spinal muscular atrophy (SMA) affects 12,000 people in the U.S. Yet, the SMA community is part of a greater community—the rare disease community—that comprises of 300 million people worldwide. Together, we create an even stronger voice to raise awareness and educate others

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Lights…Camera…Action! Search Is On for Child with SMA to Appear in NBC Television Pilot

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Cure SMA is excited to support casting directors from NBC as they search for boys and girls from the SMA community who are interested in playing a character with spinal muscular atrophy in an upcoming 20th Century Fox pilot. The team is currently focusing their search on children aged 7-11 years of age in the Chicagoland

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Genentech’s Risdiplam Showed Significant Improvement in Motor Function in People Aged 2-25 Years with Type 2 or Type 3 SMA

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Genentech, a member of the Roche Group, today presented 1-year data from the pivotal Part 2 of the SUNFISH study, evaluating risdiplam in people aged 2-25 years with Type 2 or Type 3 spinal muscular atrophy (SMA). The study showed that change from baseline in the primary endpoint of the Motor Function Measure 32 scale

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Cure SMA Adds New Equipment Item for Teens and Adults with SMA

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Along with funding SMA research and offering clinical care services, Cure SMA provides thousands of affected individuals and families with vital support and resources that help them navigate daily life with SMA. We are thrilled to add to our equipment pool inventory the Panthera S3 Swing—a lightweight manual wheelchair for adults and teens living with

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Genentech’s Risdiplam Meets Primary Endpoint in Pivotal FIREFISH Trial in Infants with Type 1 SMA

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Genentech, a member of the Roche Group, today announced positive topline results from the pivotal Part 2 of the FIREFISH study, evaluating risdiplam in infants aged 1-7 months with Type 1 spinal muscular atrophy (SMA). The primary outcome measure of the study was the proportion of infants sitting without support for at least five seconds

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Annual SMA Conference Registration Continues to Grow!

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The 2020 Annual SMA Conference registration numbers are growing quickly! We expect the 2020 conference – which includes both the Family Conference and the Research & Clinical Care Meeting – will be one of our biggest ever. Hotel rooms in our block are filling up, so be sure to register and make your hotel reservations

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Community Statement from Scholar Rock Regarding SRK-015

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Scholar Rock, a clinical-stage biopharmaceutical company focused on the treatment of serious diseases in which protein growth factors play a fundamental role, today highlighted key accomplishments of the past year and announced R&D priorities for 2020. Below please find a community statement from Scholar Rock specifically discussing SRK-015. ______________________________________________________________________________ Dear SMA Community, We at Scholar

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