Clinical Trials
Novartis Data Demonstrate Age-Appropriate Development When Zolgensma Is Used Presymptomatically and Rapid, Clinically Meaningful Efficacy in Symptomatic Children
Novartis recently announced data that reinforce the transformational benefit of Zolgensma® (onasemnogene abeparvovec), an essential, one-time treatment and the only gene therapy for spinal muscular atrophy (SMA). […]
Read More ›Community Statement from Novartis Gene Therapies
Dear SMA Community, Novartis Gene Therapies is committed to working with our partners in the SMA community to make a difference in the diagnosis, treatment, […]
Read More ›Genentech Shares New Evrysdi Data on Improved Motor Function and Survival in Babies with SMA Type 1
Genentech, a member of the Roche Group, has shared new 2-year data from Part 2 of FIREFISH, a Phase 2/3 global study evaluating Evrysdi (risdiplam) […]
Read More ›Biogen Shares New Spinraza Data Exploring Opportunities to Improve Outcomes for SMA Patients
Biogen has shared new data from the Spinraza (nusinersen) clinical development program aimed at optimizing outcomes for people with SMA and advancing understanding of the […]
Read More ›Scholar Rock Community Statement on TOPAZ 12-Month Topline Data
Scholar Rock provided the below community statement on data for apitegromab (SRK-015) following the distribution of this company press release. Dear Members of the SMA […]
Read More ›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 […]
Read More ›Research Updates from Scholar Rock
Scholar Rock Presents TOPAZ Interim Analysis Data for Apitegromab in SMA Scholar Rock recently shared 6-month interim analysis data from the TOPAZ Phase 2 clinical […]
Read More ›Evrysdi Research Updates from Genentech
New 2-year data show Evrysdi continues to demonstrate improvement or maintenance of motor function in people aged 2-25 years with Type 2 or 3 SMA […]
Read More ›Zolgensma Research Updates from Novartis Gene Therapies
New Zolgensma data demonstrate age-appropriate development when used early, real-world benefit in older children, and durability 5+ years post-treatment Novartis Gene Therapies recently shared new […]
Read More ›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 […]
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