Biogen Provides Community Statement and Q&A on the Approval of Spinraza for SMA

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Biogen provided the following statement on the approval of Spinraza for SMA: Dear Members of the SMA Community, Today, the SMA community achieved a goal that would not have been possible without the support and dedication of the families, physicians and researchers who have passionately pursued a treatment for Spinal Muscular Atrophy (SMA). We are […]

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Ionis Pharmaceuticals Issues Community Statement on the Approval of Spinraza

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Ionis Pharmaceuticals has issued the following community statement on the approval of Spinraza for SMA: Dear Members of the SMA Community, Today is a historic day for the SMA community with the approval of SPINRAZA™ (nusinersen) by the U.S. Food and Drug Administration (FDA) for the treatment of Spinal Muscular Atrophy (SMA). This is a

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FDA Approves Spinraza for SMA

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On December 23, the FDA announced that it has approved SpinrazaTM (nusinersen) to treat spinal muscular atrophy, making it the first-ever FDA-approved therapy for SMA. We are thrilled to see our community’s efforts culminate in the approval of Spinraza: not only the first-ever approved treatment for this disease, but also one that addresses the underlying

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Cure SMA Participates in a Special FDA Listening Session

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Last week, Cure SMA participated in a listening session with FDA Commissioner Robert M. Califf, MD, MACC. The FDA organized the listening session in order to gain a better understanding of the current goals, priorities, and challenges of our community. Representatives from Cure SMA presented to the Commissioner and a panel of more than 25

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Cure SMA Announces Additional Conference Support for Newly Diagnosed Families

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Thanks to the generous funding provided by the Erin Trainor Memorial Fund, Cure SMA is now offering additional support for newly diagnosed families to attend the conference. New for this year, the scholarship will now cover three hotel nights, in addition to registration fees for up to four family members, to help these families attend

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SMA Community Conducts Outreach Visits with Members of Congress

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On November 30, members of the SMA community met with 17 different legislative offices— eight from the Senate and nine from the House of Representatives.  These Senators and Representatives serve on committees important to our community’s goals, including the House Energy & Commerce Committee; Senate Health, Education, Labor and Pensions (HELP) Committee; the House and

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Jadon’s Hope Foundation Donates 15 Items to Cure SMA Equipment Pool

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Cure SMA recently recieved a donation of nine special tomato feeder seats and six telescopic ramps from Jadon’s Hope Foundation. The feeder seats and ramps were immediately sent out to families who were on a waiting list for either item. Jadon’s Hope Foundation was created by Tony and Kristin Burks, in honor of their son, Jadon,

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The Lancet Publishes Data from Phase 2, Open-Label Trial of Spinraza (Nusinersen) in Infants

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Today, Lancet published results from the Phase 2, open-label trial of nusinersen (Spinraza) in infants. This trial initiated in 2013, and patients continue to receive the drug.  The results show that infants as young as five weeks old with the most severe form of spinal muscular atrophy (SMA) – a leading genetic cause of infant

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The Miller McNeil Woodruff Foundation Makes a Generous Contribution to Cure SMA

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The Miller McNeil Woodruff Foundation awarded $87,000 to Cure SMA, in order to further research that will lead to a treatment and cure for SMA. Their gift will also help fund continued advocacy and awareness intiatives. The generous donation is made each year by Patrick and Meredith Woodruff, in honor of thier son, Miller, who passed away from

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Cure SMA-Funded Researcher, Chad Heatwole, Publishes Paper on Clinical Trial Outcomes in Adults with SMA

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Cure SMA-funded researcher, Chad Heatwole, and his team, have published a paper, “What Matters Most: A Perspective From Adult Spinal Muscular Atrophy Patients”. This project was a pilot study funded by a grant from Cure SMA, and focused on developing a reliable method for adults with SMA to report on clinical trail outcomes that are

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