Family Friendly Research Poster Session Presenters Announced

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The Family Friendly Research Poster Session will be held on Saturday, June 29th from 6:30pm to 8:00pm at the 2019 Annual SMA Conference.  The Family Friendly Research Poster Session allows for one-on-one interactions between families and researchers. Cure SMA has invited researchers, who are attending SMA Researcher Meeting, to present family friendly research posters. During […]

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Biogen Issues Q2 Community Statement on Spinraza

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Dear members of the SMA community, We are grateful for the opportunity to continue to help support individuals with spinal muscular atrophy (SMA), their caregivers and families. We deeply appreciate our shared goals and are dedicated to helping people with SMA, of all ages, and welcome the addition of new treatments in order to provide

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Team Cure SMA Celebrates Global Running Day!

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Happy Global Running Day! Is Global Running Day a made-up holiday? Kind of. But unlike other made-up holidays dedicated to eating pancakes or taking selfies, Global Running Day has the purpose of celebrating running and encouraging people to get active. Today, we’d love for you to celebrate Global Running Day by joining or supporting Team

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Scholar Rock Announces Positive Final Results from Phase 1 Clinical Trial of SRK-015 in Healthy Volunteers

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Scholar Rock today announced positive final top-line results from the Phase 1 clinical trial of its product candidate, SRK-015, a highly specific inhibitor of myostatin activation, in healthy adult volunteers. Consistent with previously announced interim findings, the final results showed robust and sustained target engagement and no apparent safety signals were observed across all tested

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Cure SMA to Host “Evening in the Park” at Disneyland Thanks to Support from AveXis

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Conference attendees will enjoy a magical “Evening in the Park” at Disneyland thanks to special support from AveXis, a Novartis company. All registered attendees for the family conference will receive (1) ticket that provides twilight entry to one of the Disneyland parks, when you check-in at registration. On Sunday, June 30th at 4:30pm, all registered

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AveXis and Cure SMA to Co-Host Webinar on the FDA’s Approval of Zolgensma

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On Wednesday, June 12th, at 10am CDT (8am PDT/9am MDT/11am EDT) AveXis, a Novartis company, and Cure SMA will co-host a webinar on the FDA approval of Zolgensma. This webinar will include information about the approval, dosing sites, future IT clinical trials, and more. The webinar will also include a Q&A session. Please note that

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AveXis Issues Community Statement on Zolgensma Approval

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Dear SMA Community, We have exciting news to share with you — a major milestone has been reached in the fight against spinal muscular atrophy (SMA)! The US Food and Drug Administration (FDA) has approved ZOLGENSMA® (onasemnogene abeparvovec-xioi) for the treatment of children less than 2 years old with SMA. This monumental day represents years

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AveXis Receives FDA Approval of Zolgensma, a Gene Therapy, for Spinal Muscular Atrophy for Patients Under Two Years of Age

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AveXis, Inc., a Novartis company, today announced that they have received FDA approval for Zolgensma (formerly AVXS-101), a gene therapy that replaces the survival motor neuron 1 (SMN1) gene, which is missing or mutated in individuals with SMA. This first approval is for intravenous (IV) delivery of gene therapy. The FDA has approved Zolgensma for

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Cure SMA Awards $150,000 Grant to Krysta Engel, PhD, University of Colorado

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Audrey Lewis founded Families of SMA, now Cure SMA, 34 years ago. Audrey recognized early on the importance of attracting new and talented researchers to SMA, with the hope that they would commit their careers to developing a treatment and cure for SMA. Cure SMA honors Audrey’s legacy with the Audrey Lewis Young Investigator Award,

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Novartis Issues Community Statement on Branaplam Clinical Study

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Dear SMA Community, We are writing to inform you that enrollment to the ongoing branaplam clinical study is now closed. Branaplam is currently being assessed for safety and efficacy in a Phase 1/2 clinical study in Type 1 infantile-onset SMA. We are pleased to announce that we have enrolled 25 infants into Part 2 of

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