Cure SMA has awarded a $150,000 research grant to Bakri Elsheikh, MD, at the Ohio State University Wexner Medical Center, for his project, “Determine the motor unit response following SMN restoration in late-onset spinal muscular atrophy”. Dr. Elsheikh and his team will study the effects of Nusinersen in adult SMA patients. By doing so, they […]
An important goal at Cure SMA is to collect data to better understand the SMA patient population, and then to report this data back to our industry partners, regulators, and payers. In collecting this data, we also hope to identify areas where additional care, advocacy, support, and research are needed. To help accomplish this goal,
Cure SMA Database Findings Published in the Journal of Neuromuscular Diseases Read More »
AveXis, Inc. and Novartis announced today that they have entered into an agreement and plan of merger. Novartis will acquire AveXis in a transaction unanimously approved by the Boards of Directors of both companies. Novartis plans a smooth transition of AveXis operations and the integration of AveXis’ talented and dedicated employees into the Novartis organization
Novartis Agrees to Acquire AveXis Read More »
The recent decision of the federal Advisory Committee on Heritable Disorders in Newborns and Children (ACHDNC) to recommend SMA for addition to the RUSP, as well as the implementation of SMA newborn screening (NBS) by several states, has ushered in the ability to identify infants with SMA before the onset of disease symptoms. To aid
SMA Newborn Screening Guidelines Published in Journal of Neuromuscular Diseases Read More »
Growing up in Virginia, Nathan Yates always had a vision for his future. From an early age, he became interested in finance, and as a teenager managed his own small portfolio of stocks. Diagnosed in 1989, he also lives with spinal muscular atrophy (SMA) Type 2. To date, Nathan’s diagnosis has never stopped him from
Community Spotlight: Nathan Yates Read More »
Cure SMA is excited to announce that AveXis is returning as a National Premier Partner for 2018. As part of their partnership, AveXis is generously supporting our Chapter Leader Summit, a full day of training and information-sharing for 70 chapter leaders from around the US, scheduled for April 2018. Many of the topics and training
Cure SMA Welcomes Back AveXis as a 2018 National Premier Partner Read More »
Cure SMA has awarded a $150,000 research grant to Brunhilde Wirth, PhD, at Institute of Cologne, Germany, for her project, “Study of combinatorial therapy based on SPINRAZA together with a novel protective genetic modifier.” Based on so-called SMA discordant families, in whom SMN1-deleted individuals remain asymptomatic, Dr. Wirth and her team have recently identified two
Cure SMA Awards $150,000 Grant to Brunhilde Wirth, PhD, Institute of Cologne, Germany Read More »
Cure SMA has awarded a $50,000 research grant to Rashmi Kothary, PhD, at the University of Ottawa, for his project, ” The shifting landscape of SMA research: towards a better understanding for a role for SMN in aging.” In this proposal, Dr. Kothary and his team seek to explore the role of SMN protein in
Cure SMA Awards $50,000 Grant to Rashmi Kothary, PhD, University of Ottawa Read More »
Scholar Rock today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to its lead antibody product candidate, SRK-015, for the treatment of spinal muscular atrophy (SMA). SRK-015 is an inhibitor of the activation of myostatin, which Scholar Rock believes has the potential to be the first muscle-directed therapy
Cure SMA has awarded a $150,000 research grant to Robin Parks, PhD, at the University of Ottawa, for his project, “Serum-derived exosomes as a biomarker for Spinal Muscular Atrophy.” Dr. Parks and his team are exploring the use of exosomes, small particles released from all cells, as potential biomarkers for SMA. They have shown that
Cure SMA Awards $150,000 Grant to Robin Parks, PhD, University of Ottawa Read More »
