Latest News
AveXis to Present Phase 1 Data at the 70th American Academy of Neurology Annual Meeting
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 […]
Read More ›SMA Community Leaders to Gather in Washington DC to Network, Learn and Advocate
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, […]
Read More ›The Discovery of Spinraza
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 […]
Read More ›There is Still Time to Complete Cure SMA’s 2018 Community Survey
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 […]
Read More ›Biogen Presents New Data at the American Academy of Neurology (AAN) Annual Meeting
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 […]
Read More ›Spring 2018 Issue of Compass Now Available Online
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 […]
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