Cure SMA Publishes Paper in Partnership with NeurologyLive on Impact of COVID-19

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    Cure SMA has published a paper, titled “Spinal Muscular Atrophy and COVID-19: Guidance and Resources for Providers,” in partnership with NeurologyLive. The article highlights findings from a recent survey conducted by Cure SMA to measure the impact of COVID-19 on the spinal muscular atrophy (SMA) patient and family community. As of June 1, […]

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Novartis Presents New Zolgensma® Data Further Demonstrating Therapeutic Benefit

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    Novartis Gene Therapies today announced new interim data from the ongoing Phase 3 STR1VE-EU clinical trial for Zolgensma® (onasemnogene abeparvovec). The data demonstrates that patients with spinal muscular atrophy (SMA) Type 1 continued to experience significant therapeutic benefit, including event-free survival, rapid and sustained improvement in motor function, and motor milestone achievement, including

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Genentech Presents New 2-Year Data for Evrysdi (risdiplam) in Infants with Type 1 SMA

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Genentech, a member of the Roche Group, today announced new 2-year data from Part 1 of the pivotal FIREFISH study of Evrysdi™ (risdiplam) in infants aged 2-7 months with symptomatic Type 1 spinal muscular atrophy (SMA). The 2-year results in infants treated with the therapeutic dose of Evrysdi (17/21) showed that they continued to improve

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Community Spotlight: Xavier and Rose’s SMA Diagnosis

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Carrie Menke is a South Dakota native who resides in Sioux Falls with her husband Tony and their four children — Xavier, Colette, Avila, and Rose. Carrie is one of the many advocates across the country who is supporting efforts to have spinal muscular atrophy (SMA) added to her state’s newborn screening panel. Her passion

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Novartis Issues Community Statement with Update on AVXS-101 Intrathecal Clinical Development Program

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Dear SMA Community, We recently received important feedback from the U.S. Food and Drug Administration (FDA) following their review of data from the STRONG study of AVXS-101 intrathecal (IT) formulation in older patients with spinal muscular atrophy (SMA) and we’d like to share an update with you.  (Please see the press release issue by Novartis

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Team Biogen Runs Over 22,000 Miles During SMA Awareness Month

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        Team Biogen hit the ground running throughout the month of August in honor of SMA Awareness Month. Together, the team logged 22,059 miles collectively from August 1st to August 31st. Nearly 300 Biogen employees came together, virtually of course, to raise funds and awareness for SMA. They successfully raised close to

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Community Spotlight: Braedon’s Newborn Screening Diagnosis

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September is Newborn Screening Awareness Month, a chance to talk about the progress we have made in getting spinal muscular atrophy (SMA) added to newborn screening panels and the work still to be done. This month, you will hear about newborn screening milestones, stories, and steps you can take to ensure all states screen newborns

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Biogen Q3 2020 Community Statement: COVID-19 Updates and Latest Milestones

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Dear Members of the SMA Community, We continue to work with the healthcare community to help people and families living with SMA get the support they need. As such, we wanted to share Biogen’s latest efforts on the following: We are committed to sharing information that will aid healthcare providers and individuals with SMA in

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Scholar Rock Announces that SRK-015 has Received Rare Pediatric Disease Designation from U.S. FDA for the Treatment of SMA

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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 announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease (RPD) designation for SRK-015 for the treatment of Spinal Muscular Atrophy (SMA), a progressive, rare genetic disease that

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Community Spotlight: Nicole Stickane Advocates for Newborn Screening in Texas

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In late July, the Texas Newborn Screening Advisory Committee held a meeting— virtually, of course —to hear an update on the status of statewide newborn screening for spinal muscular atrophy (SMA). State officials shared updates and members of the SMA community shared public comments. Nicole Stickane shared her family’s experience in support of adding SMA

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