Cure SMA Coverage and Payment Policy Project Continues to Move Forward

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The Cure SMA Coverage and Payment Policy Project has two distinct but complementary tracks: Outreach on behalf of our community. We’re working with public and private insurers, regulators, and other stakeholders, advocating for prompt, comprehensive coverage for SMA treatments and care.  Outreach to our community. We’re working to provide the necessary tools and information so […]

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Genentech Roche Releases Community Letter with Clinical Trial Updates

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Genentech Roche provided the following community statement with clinical trial updates for SUNFISH (Type 2/3), FIREFISH (Type 1) and JEWELFISH. Dear SMA community, At Genentech (A Member of the Roche Group) we are committed to addressing the urgent needs of people living with SMA. We are happy to share this update on our two investigational

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Cure SMA Releases Schedule for the 2017 Annual SMA Conference

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Cure SMA has released the schedule for the 2017 Annual SMA Conference, to be held at Walt Disney World in Orlando, FL, June 29 – July 2. The schedule includes the timeline of workshops and events, including the Meet and Greet, Researcher Relay Race, Teen Social, Newly Diagnosed Program, Family Friendly Poster Session, PJ Party

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Cure SMA Awards $140,000 Grant to Joceyln C̫t̩, PhD, University of Ottawa

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Cure SMA has awarded a $140,000 research grant to Joceyln Côté, PhD, at The University of Ottawa, for his project, “Investigating the mechanism by which SMN regulates translation: identification of novel therapeutic targets.” Understanding the function of the SMN protein is vital to understanding SMA and serves to aid in the development of therapeutics. In

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American College of Obstetricians and Gynecologists Recommends Expanded SMA Carrier Screening

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The American College of Obstetricians and Gynecologists has released updated guidance on carrier testing for genetic conditions. The updated guidance includes, for the first time, a recommendation that all women who are pregnant or considering pregnancy be screened for spinal muscular atrophy. Previously, carrier screening was only recommended for women with a known family history

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AveXis Reports Results from Phase 1 Trial of AVXS-101

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AveXis, Inc., a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, today reported topline results from the Phase 1 trial of AVXS-101 in spinal muscular atrophy (SMA) Type 1. The company also reported financial results for the fourth quarter and full year ended December 31, 2016, recent

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Know Your Resources: How Congressional Caseworkers Can Help You Obtain Insurance Coverage for New Therapies

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Your elected officials who represent you in Washington, D.C. have caseworkers in their regional and district offices in your communities. These caseworkers can help you work with and better understand government programs, like Medicaid, Medicare, TRICARE, and Social Security. Caseworkers, also sometimes called constituent services representatives, are in the local community offices of federal elected

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Cure SMA Awards $140,000 Grant to Arthur Burghes, PhD, The Ohio State University

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Cure SMA has awarded a $140,000 research grant to Arthur Burghes, PhD, at The Ohio State University, for his project, “Identification of SMA modifiers and deletion/duplication junctions in the SMA region.” All individuals with SMA lack the SMN1 gene, but have one or more copies of SMN2, the SMA “backup gene.” In general, those with

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Cure SMA Releases Care Series Booklet on Insurance

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Cure SMA is excited to announce the launch of our newest care series booklet, Choice and Connection to Care: A Health Insurance Roadmap for People Living with Spinal Muscular Atrophy (SMA) and Their Caregivers. You can download a copy of this booklet now from our website, or send an email to [email protected] to request a

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SMA is Submitted to the Federal Newborn Recommended Uniform Screening Panel (RUSP)

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Cure SMA and the SMA Newborn Screening Coalition are excited to announce we have completed the submission of SMA to the Recommended Uniform Screening Panel (RUSP). Administered by the Secretary’s Advisory Committee on Heritable Disorders in Newborns and Children (SACHDNC), part of the Department of Health and Human Services, the RUSP is a list of

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