Dear Members of the SMA Community, For the past several years, we’ve been working to collect data and information on our community’s experiences, goals, hopes, and challenges. We know that the voice of our community is powerful. By sharing our stories, we can communicate our priorities to the FDA and regulators, provide insight into daily […]
There is Still Time to Complete Cure SMA’s 2018 Community Survey Read More »
Biogen recently presented new data on Spinraza for the treatment of spinal muscular atrophy (SMA) during the 70th American Academy of Neurology (AAN) Annual Meeting, currently taking place from April 21-27 in Los Angeles, California. Both platform and poster presentations highlighted the benefits Spinraza provides for individuals with spinal muscular atrophy (SMA) across the age
Biogen Presents New Data at the American Academy of Neurology (AAN) Annual Meeting Read More »
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 »
