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 »
Dear Members of the SMA Community, Each year, the entire rare disease community comes together on the last day of February to recognize and raise awareness for the 300 million people worldwide, including 25-30 million Americans, impacted by a rare disease like SMA: that is one in 10 Americans, and more than
Biogen SMA Q1 2021 Community Statement Read More »
Genentech, a member of the Roche Group, announced in a press release that Evrysdi™ (risdiplam) data from the dose finding Part 1 of the pivotal FIREFISH study in infants with symptomatic Type 1 spinal muscular atrophy (SMA) were published in the February 24, 2021 online issue of the New England Journal of Medicine (NEJM). The data show
Results from Genentech’s Evrysdi Study in Infants with Type 1 SMA Published in NEJM Read More »
Adults with spinal muscular atrophy (SMA) are invited to take advantage of Cure SMA’s latest support resource, the Adults with SMA Virtual Therapy Program, which has been generously sponsored by Genentech and the Dhont Family Foundation. The Adults with SMA Virtual Therapy Program provides adults with SMA three complementary, 30-minute live video sessions with a
Cure SMA Launches Virtual Therapy Program for Adults with SMA Read More »
Over the past year and a half, we hope that you have found this page to be a helpful resource as you navigated the COVID-19 pandemic. As we seek to come out the other side and begin to re-engage with our family, friends, and communities, know that Cure SMA’s work for the SMA community continues.
Cure SMA Coronavirus (COVID-19) Information Center Read More »
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 to achieve new developmental milestones. However, these treatment advances have also raised new questions for the community, including whether combining treatments is safe and offers additional efficacy. To better understand the
Cure SMA Offer Perspectives on Combination Therapy for SMA Read More »
