March 2021

Cure SMA-Funded Researcher, Chad Heatwole, Publishes Paper on SMA-HI, a Clinical Trial Outcome in Adults with SMA

Dr. Chad Heatwole, a Cure SMA-funded researcher, and his team have published a paper in the Journal, Muscle Nerve, titled “The Spinal Muscular Atrophy Health Index (SMA-HI): A Novel Outcome for Measuring How a Patient Feels and Functions.” This project, funded by Cure SMA, focused on developing a multi-faceted, disease-specific, patient-reported outcome to measure an SMA […]

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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 clinical trials. Oral presentation details: Title: Predictive Factors of Nusinersen Treatment Response in Infantile-onset SMA: Results from the ENDEAR/SHINE Studies Presenter: Laurent Servais, MD, PhD Title: Nusinersen in Children with SMA Who

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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 trial evaluating the potential of apitegromab, a highly selective inhibitor of latent myostatin, in improving motor function in patients with Types 2 and 3 spinal muscular atrophy (SMA). A this

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Community Statement from Genentech

Dear SMA Community, Based on our commitment to the SMA community to provide transparent, timely updates related to our medicines, we are writing to share information regarding recent reports of leaking bottles of Evrysdi, and provide information on steps we are initiating as a result. Below we have provided additional details and next steps should

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Community Statement from Novartis Gene Therapies

Dear Cure SMA Community, Novartis Gene Therapies is committed to patient safety and the ongoing monitoring of adverse events as it relates to the use of ZOLGENSMA® (onasemnogene abeparvovec-xioi). As with all commercially marketed products, we are continuously monitoring the safety of ZOLGENSMA, and sharing safety data as part of the required pharmacovigilance with health

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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 Genentech, a member of the Roche Group, recently shared new exploratory 2-year, longer-term data from Part 2 of SUNFISH, a global placebo-controlled study evaluating Evrysdi (risdiplam) in people aged 2-25

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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 data that reinforce the transformational benefit of Zolgensma (onasemnogene abeparvovec), a one-time treatment for spinal muscular atrophy (SMA). The overall safety profile remains favorable following pre-symptomatic treatment, in the long-term

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Cure SMA Publishes Quality of Life in Teens and Young Adults Manuscript in Orphanet Journal of Rare Diseases

Current knowledge regarding clinical meaningfulness and quality of life amongst teens and young adults with spinal muscular atrophy (SMA) is limited. Much of the available qualitative data on this population has been obtained from the perspective of the parents/caregivers rather than the individuals themselves. Knowing this, Cure SMA is pleased to announce the publication of a manuscript,

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