Author name: Sarah McCall

AveXis, a Novartis company, announced a one-time infusion of Zolgensma® (onasemnogene abeparvovec-xioi) showed rapid, significant, and clinically meaningful therapeutic benefit in patients with spinal muscular atrophy (SMA) across a range of studies, including in patients treated pre-symptomatically, and sustained durability in patients now up to 5 years post-dosing and some patients more than 5 years […]

Cure SMA is pleased to announce the release of “Spotlight on SMA: The Spinal Muscular Atrophy Treatment Landscape,” the final supplement in a two-part series developed in collaboration with Neurology Reviews. The goal of this partnership is to increase awareness of SMA amongst healthcare providers, including current clinical guidelines and the need for prompt diagnosis

This article is part of a series of Cure SMA grant announcements being shared throughout the winter/spring. Cure SMA has awarded a $150,000 grant to Emily Welby, Ph.D., of the Medical College of Wisconsin for her project titled, ” The role of astrocytes in SMA motor neuron synapse defects.” Dr. Welby is a post-doctoral researcher working

This article is part of a series of Cure SMA grant announcements being shared throughout the winter/spring. Cure SMA has awarded a $190,000 research grant to Umrao Monani, Ph.D., of Columbia University, for his project titled, “Genetic suppressors of the SMA phenotype.” Dr. Monani is associate professor of Neurology and Pathology & Cell Biology, and

This article is part of a series of Cure SMA grant announcements being shared throughout the winter/spring. Cure SMA and Cure SMA Canada have awarded a $100,000 research grant to Rashmi Kothary, Ph.D., at the Ottawa Hospital Research Institute, for his project titled, “Characterization of canonical disease features in a novel mouse model of SMA