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 Community, We are pleased to share 12-month topline results from our TOPAZ study, a Phase 2 clinical trial to evaluate the safety and efficacy of apitegromab (SRK-015) in individuals with […]
Scholar Rock Community Statement on TOPAZ 12-Month Topline Data Read More »
In honor of National Volunteer Month in April, Cure SMA is sharing stories and quotes from volunteers who support our programs and further grow our community. Cure SMA is incredibly grateful for the volunteers who keep this organization strong. You might know Nick Farrell as the newest Chairperson of the Cure SMA Board of Directors.
Community Spotlight: Nick Farrell Read More »
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
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
Nusinersen Research Updates Presented by Biogen Read More »
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
Research Updates from Scholar Rock Read More »
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
Community Statement from Genentech Read More »
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
Community Statement from Novartis Gene Therapies Read More »
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
Evrysdi Research Updates from Genentech Read More »
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
Zolgensma Research Updates from Novartis Gene Therapies Read More »
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,
