Search Results: spinraza

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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Cure SMA Offer Perspectives on Combination Therapy for SMA

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New developments in treatments for spinal muscular atrophy (SMA) over the last few years have changed what is possible for people with SMA, allowing them […]

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Cure SMA’s New Year Letter for 2021

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Dear SMA Community, While 2020 did not pan out as we had expected, Cure SMA remains extremely optimistic about the future for our community in […]

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Biogen Provides SMA Community Update on DEVOTE Study

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Below we have provided an update from Biogen regarding the status of its DEVOTE clinical trial. For more information about SPINRAZA (nusinersen), visit https://www.curesma.org/spinraza/. ++++++++++++++++++++ […]

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