November 2019

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

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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 several others in clinical trials. The advent of these new treatments for SMA has led to community discussion around the possibility of combining therapies in the hopes of better outcomes. […]

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9th Annual “Hope on the Hill” Congressional Dinner

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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, government officials, and industry leaders will gather to discuss advancements in research, treatment, and patient care, as well as harness the latest momentum to set the course for the next

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

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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) to attend the 2020 Annual SMA Conference. The conference will take place June 11-June 14, 2020, at Disney’s Yacht & Beach Club Resort in Orlando, FL. Thanks to a generous

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

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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 granted Priority Review for risdiplam, an investigational survival motor neuron-2 (SMN-2) splicing modifier for spinal muscular atrophy (SMA). If approved, risdiplam, an orally administered liquid, would be the first at-home

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SMA Concordance Among Siblings Published in the Journal of Neuromuscular Diseases

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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 Cure SMA Cohort.” This paper examined the concordance and discordance of SMA sub-type among siblings in order to learn about the family experience of SMA, support the identification of genetic

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Caring for the Caregiver by Brynne Willis

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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 giving, as well as the one receiving. Despite this, the extra demands placed on caregivers can often lead to chronic stress and stress-related disorders. Chronic stress, if left untreated, can

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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

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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 with spinal muscular atrophy (SMA). The planned preliminary PK/PD analysis, which includes data from 29 patients with SMA across all three cohorts, showed dose-proportional drug exposure and demonstrated target engagement, as

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

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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, 2019. The all-star team included: David Alexander, Jose Ricardo Alverde, Jillian Ament, Michela Bassano, Sarah Bromley, Amanda Dinan, Emma Fazio, Sarah Fazio, Emily Gross, Christine Guiney, Danielle Joslin, Taline Lahcanski,

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Genentech’s Risdiplam Meets Primary Endpoint in Pivotal SUNFISH Trial in People With Type 2 or 3 Spinal Muscular Atrophy

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Genentech, a member of the Roche Group, today announced positive data from the pivotal Part 2 of the SUNFISH study evaluating risdiplam in people aged 2-25 years with Type 2 or 3 spinal muscular atrophy (SMA). The study met its primary endpoint of change from baseline in the Motor Function Measure 32 (MFM-32) scale after

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