Richard Finkel, M.D., the chief of neurology at Nemours Children’s Hospital in Orlando, Fla., was honored with the American Academy of Neurology’s 2018 Sidney Carter Award in Child Neurology, in recognition of his achievements in treating children with muscular dystrophy (MD) and spinal muscular atrophy (SMA). Dr. Finkel’s presentation in receipt of the Sidney Carter […]
The Phase 1, open-label, dose-escalation trial was designed to evaluate the safety and tolerability of AVXS-101 in patients with SMA Type 1. The key measures of efficacy were the time from birth to an event and video confirmed achievement of ability to sit unassisted. Additionally, several exploratory objective measures were assessed, including a standard motor
AveXis to Present Phase 1 Data at the 70th American Academy of Neurology Annual Meeting Read More »
On Thursday, April 26, more than 70 chapter leaders, event organizers and board/committee members, representing 24 states and DC, will gather in Washington DC for two days of networking, learning, and advocacy. The activities include a day of advocacy on Capitol Hill, meeting with House and Senate offices, and a day of training and information
SMA Community Leaders to Gather in Washington DC to Network, Learn and Advocate Read More »
SMA is caused by a mutation in the survival motor neuron gene 1 (SMN1). In a healthy person, this gene produces a protein—called survival motor neuron protein or SMN protein—that is critical to the function of the nerves that control our muscles. Without it, those nerve cells cannot properly function and eventually die, leading to
The Discovery of Spinraza Read More »
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
