Search Results: spinraza

Drug Discovery

Make today a breakthrough. Drug discovery takes what we’ve learned about the causes and biology of spinal muscular atrophy (SMA) and utilizes it for basic […]

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Positive New Data for Genentech’s Evrysdi in Largest Trial Ever Undertaken in Patients With Previously-Treated Spinal Muscular Atrophy (SMA)

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Genentech, announced earlier this month new two-year data from the JEWELFISH study evaluating Evrysdi® (risdiplam) in people with Type 1, 2 or 3 SMA aged 6 […]

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Biogen Exercises Option with Ionis to Develop and Commercialize Investigational ASO for SMA

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      Biogen Inc. and Ionis Pharmaceuticals, Inc., recently announced that Biogen exercised its option to obtain from Ionis a worldwide, exclusive, royalty-bearing license […]

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Cure SMA Releases Updated Drug Pipeline

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We’ve recently released an update to the SMA drug pipeline. This latest version includes: 20 active programs, including three approved therapies. 10 pharmaceutical partners. 12 […]

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Scholar Rock Shares SMA Community Letter and Announces Design of Phase 3 SAPPHIRE Clinical Trial

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Dear SMA Community, We are excited to announce the design of SAPPHIRE, a Phase 3 clinical trial to further evaluate the safety and efficacy of […]

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Community Statement from Biogen: New Clinical Study Planned to Evaluate the Potential Benefit of an Investigational Higher Dose of Nusinersen in People with SMA Previously Treated with Evrysdi® (risdiplam)

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Biogen is committed to improving health outcomes for people impacted by spinal muscular atrophy (SMA) and generating data to assess if there are treatment pathways […]

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Cure SMA Convenes Working Group to Discuss Biomarkers for SMA

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The advent of three approved SMN-upregulating therapeutics for spinal muscular atrophy has dramatically changed the natural history of the disease. There are, however, many uncertainties […]

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Thank You for Another Fantastic Virtual SMA Conference!

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Cure SMA would like to thank everyone who attended the recent 2021 Virtual SMA Conference and 2021 Virtual SMA Research & Clinical Care Meeting. It was an impactful […]

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Nusinersen Research Updates Presented by Biogen

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Biogen recently presented data about ongoing studies of SPINRAZA (nusinersen) in patients with Spinal Muscular Atrophy. Updates included the NURTURE, ENDEAR, SHINE, DEVOTE, and RESPOND […]

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Biogen SMA Q1 2021 Community Statement

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      Dear Members of the SMA Community, Each year, the entire rare disease community comes together on the last day of February to […]

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