June 2017

Cure SMA Funded Standard of Care Paper Published in Neuromuscular Disorders Journal

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The journal Neuromuscular Disorders has published a paper reporting on the findings of an international workgroup convened to update the Standard of Care (SOC) for SMA. Cure SMA provided funding support for this project, and Mary Schroth, medical director for Cure SMA, served on the workgroup. A SOC for SMA was first issued in 2007, […]

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The 2017 Annual SMA Conference is Here!

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The 2017 Annual SMA Conference, now in its 29th year, is set to kick off on June 29 and continue into a weekend full of programs and events that bring families, researchers and healthcare professionals together. This year’s conference has already surpassed the records of previous years. We’re expecting over 2,000 attendees, including almost 450

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Call-to-Action: Senate Health Care Bill Released and Vote Expected Next Week

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Yesterday, members of the Senate released draft text of their much awaited legislation called the Better Care Reconciliation Act (“Better Care Act”) of 2017. As you’ll recall from previous updates (links here and here), the Senate has been working to put together its own legislation to modify key parts of the Patient Protection and Affordable

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Launch of Phase 2 SMA Industry Collaboration Announced

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Cure SMA is pleased to announce the launch of Phase 2 of our SMA Industry Collaboration. The SMA Industry Collaboration is a multi-faceted partnership that brings together pharmaceutical companies, Cure SMA, and other nonprofit organizations, to share information, ideas, and data. The SMA Industry Collaboration works together to address scientific, clinical and regulatory topics that

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2017 Annual SMA Conference Booklet Now Posted

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The 2017 Annual SMA Conference Booklet is now posted. The booklet includes: A complete schedule with information about the special events we’ve got planned including: the Family Friendly Research Poster Session, Saturday Night at Magic Kingdom Park and Sunday’s Newborn Screening Symposium Information on Saturday afternoon panels focusing on SMA drugs in development and drug

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SMA Newborn Screening Moves Forward in Missouri and Florida

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The spinal muscular atrophy community is celebrating significant progress toward adding SMA to newborn screening panels in two states. Missouri to Become the First State to Screen for SMA In Missouri, Senate Bill 50 has been passed by both the Missouri House and Missouri Senate, and is currently awaiting the governor’s signature. This bill would

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AveXis Announces Alignment with the FDA on Company’s GMP Commercial Manufacturing Process for AVXS-101

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AveXis today announced alignment with the U.S. Food and Drug Administration (FDA) on the company’s Good Manufacturing Practice (GMP) commercial manufacturing process for AVXS-101 following the receipt of minutes from the Type B Chemistry Manufacturing and Controls (CMC) meeting. This alignment includes support for the proposed commercial manufacturing process, the proposed analytical methods and corresponding

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Cure SMA Advocacy Activities for the 2017 Year-to-Date

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The first half of 2017 has been an active six months for Cure SMA and SMA advocates. Cure SMA and members of our community have been working with stakeholders groups across multiple settings to ensure our patients’ voices are heard. Below is a summary of the key issues and audiences we’ve targeted in our engagement

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Update: President’s Budget Proposes Significant Cuts to Federal Health Programs That Are Critically Important to SMA Patients and Families

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The President recently released his detailed budget proposal for Fiscal Year 2018, which proposed significant funding cuts to numerous federal programs, including many that have a direct impact on the health and well-being of individuals and families affected by SMA. The federal Fiscal Year runs from October 1, 2017 through September 30, 2018. Of serious

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