Author name: Sarah McCall

Cure SMA Announces Expanded Phase 3 of Real World Evidence Collaboration

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Cure SMA is pleased to announce the launch of an expanded Phase 3 of the Real World Evidence Collaboration. The collaboration was established to leverage the experience, expertise and resources of pharmaceutical and biotechnology companies and nonprofit organizations involved in development of SMA therapeutics to guide the future direction of real world evidence collection and […]

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New DOT Rule Will Require Truly Accessible Lavatories on New Single Aisle Aircraft

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On the anniversary of the Americans with Disabilities Act, the U.S. Department of Transportation (DOT) announced a final rule that will require airlines to take short- and long-term action to make lavatories on new single-aisle aircraft more accessible for people with disabilities, including those who fly in a wheelchair. “The inability to safely access and

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SMA Scientists and Clinicians from Around the World Gather at the 27th Annual SMA Research & Clinical Care Meeting

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At the end of June, SMA researchers and clinicians from around the world met in Orlando, Florida, for the 2023 Annual SMA Research & Clinical Care Meeting.   The annual meeting provides an opportunity for SMA scientists and clinicians to share their most recent discoveries and learnings, and is an incubator for collaborations, including cross-disciplinary

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Thank You for an Amazing 2023 Annual SMA Conference!

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Thank you to everyone who attended the 2023 Annual SMA Conference at Disney’s Yacht & Beach Club in Orlando, Florida! It was an impactful weekend of opportunities to connect, learn, and have fun. We are happy to announce that, including on-site registration, there were over 3,200 attendees in total – a record for the Annual

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Living Unlimited with Spinal Muscular Atrophy: A Booklet for Adults with SMA

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Cure SMA is excited to announce our newest publication geared toward adults with SMA. Thanks to contributions and feedback from adults with SMA in our community, we have collectively pulled together this booklet on adult-specific topics of importance. Check out the booklet here! Topics covered in Living Unlimited with SMA include: Planning for your Future

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Cure SMA Applauds Federal Agency Decision that Should Lead to Better Insurance Coverage for Seat Elevation Systems in Power Wheelchairs

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Thanks to the advocacy of individuals with spinal muscular atrophy (SMA) and others, the Centers for Medicare and Medicaid Services (CMS) now considers seat elevation systems in power wheelchairs as being “reasonable and necessary” for purposes of Medicare coverage. The federal agency updated its national policy to allow Medicare coverage for seat elevation systems in

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Biogen Announces New Updates Across its SMA Research Program at 2023 MDA Conference

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First patient treated in the ASCEND study evaluating the potential benefit of investigational higher dose nusinersen in children, teens and adults previously treated with Evrysdi® (risdiplam) Baseline characteristics indicate all nine infants and toddlers enrolled in RESPOND had suboptimal clinical status in ≥2 areas after receiving Zolgensma® (onasemnogene abeparvovec); there were no new safety findings with subsequent

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Novartis shares Zolgensma long-term data demonstrating sustained durability up to 7.5 years post-dosing; 100% achievement of all assessed milestones in children treated prior to SMA symptom onset

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Children in LT-001 treated after SMA symptom onset maintained or achieved additional milestones up to 7.5 years post one-time intravenous infusion All children (100%) in the presymptomatic intravenous cohort of LT-002 maintained or achieved all assessed motor milestones, including independent walking To date, more than 3,000 children with spinal muscular atrophy have been treated with

Novartis shares Zolgensma long-term data demonstrating sustained durability up to 7.5 years post-dosing; 100% achievement of all assessed milestones in children treated prior to SMA symptom onset Read More »

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