The Spring 2018 issue of Compass is now available online. The issue covers Cure SMA’s newborn screening efforts, including how NBS can help children diagnosed with SMA, the addition of SMA to the federal Recommended Uniform Screening Panel (RUSP), and state and federal advocacy efforts. For a condition like SMA to become part of routine
Spring 2018 Issue of Compass Now Available Online Read More »
The deadline to register for the 2018 Annual SMA Conference is just two weeks away! Registration will close on Friday, May 4th. Over 1,100 people have already registered – don’t miss your chance to engage with families, researchers and medical providers at largest SMA conference in the world! Register The conference will be filled with
Registration for the 2018 Annual SMA Conference Closes in Two Weeks Read More »
Genentech/Roche announced today that new data on its approved and investigational medicines for neurological conditions will be presented during the 70th American Academy of Neurology (AAN) Annual Meeting from April 21-27 in Los Angeles, California. The data will include investigational research from Genentech on drugs in development for spinal muscular atrophy (SMA). The SMA presentations
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 »
