Cure SMA Awards $150,000 Grant to Umrao Monani, PhD, Columbia University

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Cure SMA has awarded a $150,000 research grant to Umrao Monani, PhD, at Columbia University, for his project, “SMA modulators as a means to revealing disease mechanisms.” Dr. Monani and his team have generated SMA model mice and shown that their genetic background can alter disease severity. By studying these mice, they have identified two […]

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Biogen Announces New Interim Phase 2 Results From NURTURE

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Biogen recently announced new interim Phase 2 results from NURTURE, the ongoing open-label, single-arm study evaluating the efficacy and safety of SPINRAZA or nusinersen among pre-symptomatic infants with spinal muscular atrophy. Additionally, data from a case series conducted on SPINRAZA-treated teens and young adults was also released. The main findings of which are summarized below:

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Cure SMA Launches Second Annual Community Survey to Address Important Issues in SMA Treatment

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Dear Members of the SMA Community, For the past several years, we’ve been working to collect data and information on our community’s experiences, goals, hopes, and challenges. We know that the voice of our community is powerful. By sharing our stories, we can communicate our priorities to the FDA and regulators, provide insight into daily

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Indiana is the Fourth State to Adopt Permanent SMA Screening

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Last night, Governor Eric Holcomb of Indiana signed HB 1017, adding spinal muscular atrophy (SMA) and severe combined immunodeficiency (SCID) to the state’s newborn screening panel. The bill makes Indiana the fourth state in the country to adopt permanent SMA screening, following Missouri, Utah and Minnesota. The legislation was sponsored by Representative Doug Gutwein, and

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Cure SMA Awards $150,000 Grant to Allison Ebert, PhD, Medical College of Wisconsin

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Cure SMA has awarded a $150,000 research grant to Allison Ebert, PhD, at the Medical College of Wisconsin, for her project, “Role of astrocyte-produced miR-146a in SMA pathology.” SMA is caused by mutations in the SMN1 gene resulting in low levels of SMN protein, which is expressed in every cell in the body. The goal

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Cure SMA Welcomes New Medical Advisory Council Committee Members

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We are excited to welcome 20 new members to our Medical Advisory Council (MAC). These clinicians represent eleven specialties, including specialties that were not previously represented on the MAC. Our new members are: Alison Ballard, RN, MSN, Pediatric Nurse Practitioner and Neuromuscular Care Coordinator at Children’s Hospital Colorado in Denver, CO  William Bell, BS Pharm,

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SMA Newborn Screening Moves Forward in Indiana, Ohio and Georgia

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The spinal muscular atrophy community is celebrating significant progress toward adding SMA to newborn screening panels in several states. Indiana Poised to Become the Fourth State to Adopt SMA Screening In Indiana, HB 1017 has been passed by both the House and Senate, and is currently awaiting Governor Eric Holcomb’s signature. This bill would institute

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#ShowYourRare and Raise SMA Awareness on World Rare Disease Day

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Rare Disease Day is today, February 28! And we’re calling on our entire spinal muscular atrophy community to get involved. Rare Disease Day is important to us because it is a time for individuals impacted by a rare disease—or those whose loved ones are impacted—to come together. It is a time for us to raise

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Last Week’s Webinar on Treatment Access and Clinical Trials Now Available

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A recording of last week’s webinar updating the community on treatment access and clinical trials is now available online. A PDF of the webinar presentation is also available for download. An update on the status of Spinraza dosing across the US included the following information: As of December 2017, nearly 2,000 US patients had been

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