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Registration Now Open for the 2020 Annual SMA Conference!

December 6, 2019
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We are excited to announce that registration is now open for the 2020 Annual SMA Conference, which will be held at Disney’s Yacht & Beach […]

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Update on Cure SMA Summit of Strength Program – Gearing Up for 2020!

December 4, 2019
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The 2019 Summit of Strength Program was a huge success, with more than 135 speakers sharing their expertise on numerous highly rated and unique topics. […]

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Check Out the Cure SMA Booklet on “Scientific Considerations for Drug Combinations”

November 27, 2019
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There are now two treatments approved by the U.S. Food and Drug Administration (FDA) that target the underlying genetics of spinal muscular atrophy (SMA) and […]

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9th Annual “Hope on the Hill” Congressional Dinner

November 26, 2019
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Just one more week until the 9th Annual “Hope on the Hill” Congressional Dinner in Washington D.C.! Next Tuesday, December 3rd, SMA families and advocates, […]

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Cure SMA and Genentech Partner to Provide Travel Support to Adults with SMA for the 2020 Annual SMA Conference

November 25, 2019
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Cure SMA is thrilled to announce that we will, once again, be offering scholarship opportunities to adults with SMA (18 years of age and older) […]

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Community Statement from Genentech: FDA Grants Priority Review to Risdiplam for Spinal Muscular Atrophy

November 25, 2019
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Genentech, a member of the Roche Group, today announced that the U.S. Food and Drug Administration (FDA) has accepted the New Drug Application (NDA) and […]

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SMA Concordance Among Siblings Published in the Journal of Neuromuscular Diseases

November 21, 2019
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Earlier this month, an article was published in the Journal of Neuromuscular Diseases titled, “Spinal Muscular Atrophy (SMA) Subtype Concordance in Siblings: Findings from the […]

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Caring for the Caregiver by Brynne Willis

November 20, 2019
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Many consider the deepest expression of love is being a caregiver. Providing for someone’s needs is incredibly special and rewarding to the one who is […]

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Scholar Rock Reports Preliminary Pharmacokinetic and Pharmacodynamic Data from TOPAZ Phase 2 Trial of SRK-015 for the Treatment of Patients with Spinal Muscular Atrophy

November 19, 2019
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Scholar Rock today announced preliminary pharmacokinetic (PK) and pharmacodynamic (PD) results from the TOPAZ Phase 2 proof-of-concept trial of SRK-015 for the treatment of patients […]

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Team Cure SMA Raises Over $45,000 in the New York City Marathon

November 18, 2019
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Fifteen Team Cure SMA runners successfully raised over $45,000 for Cure SMA while running in the 2019 TCS New York City Marathon on November 3, […]

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