Search Results: Spinraza
Positive New Data for Genentech’s Evrysdi in Largest Trial Ever Undertaken in Patients With Previously-Treated Spinal Muscular Atrophy (SMA)
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 […]
Biogen Exercises Option with Ionis to Develop and Commercialize Investigational ASO for SMA
Biogen Inc. and Ionis Pharmaceuticals, Inc., recently announced that Biogen exercised its option to obtain from Ionis a worldwide, exclusive, royalty-bearing license […]
Cure SMA Releases Updated Drug Pipeline
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 […]
Scholar Rock Shares SMA Community Letter and Announces Design of Phase 3 SAPPHIRE Clinical Trial
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 […]
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)
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 […]
Cure SMA Convenes Working Group to Discuss Biomarkers for SMA
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 […]
Thank You for Another Fantastic Virtual SMA Conference!
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 […]
Nusinersen Research Updates Presented by Biogen
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 […]
Biogen SMA Q1 2021 Community Statement
Dear Members of the SMA Community, Each year, the entire rare disease community comes together on the last day of February to […]
Cure SMA Offer Perspectives on Combination Therapy for SMA
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 […]