AveXis Announces Launch of New Patient-Oriented Website Focused on Spinal Muscular Atrophy

Dallas, TX, December 04, 2014 --(PR.com)-- AveXis, Inc., a biotechnology company focused on using gene therapy to transform the lives of patients with severe genetic and orphan diseases like Spinal Muscular Atrophy (SMA), is pleased to announce the launch of the company’s brand new website specifically focused on SMA: www.smastudy.org. The December 2014 unveiling of the website is an integral element to AveXis’ multi-pronged effort to grow awareness of the ongoing and upcoming clinical trials in SMA.

In contrast to the company’s corporate website centered on AveXis, the new SMA-focused website is designed to communicate directly with parents of children with SMA who are looking for options to combat SMA.

The new website offers information about the ongoing open enrollment in the Gene Transfer Clinical Trial for SMA Type 1 (NCT02122952), information about the gene therapy biologic currently called chariSMA, and links to connect patients, parents, and families with organizations, foundations, and support and advocacy groups around the world. The website also provides links to other available clinical trials for patients suffering from all types of SMA.

“It is very important for AveXis to remain rooted and communicate effectively to the community which is so supportive of our work in gene therapy. The intention of this new website is to empower people around the world to learn about SMA, to inform them of their clinical trial options, and to provide links to educational support organizations, which are always useful when looking for answers,” stated John A. Carbona, Chief Executive Officer of AveXis.

To see the new patient-oriented, SMA-focused website, please visit: www.smastudy.org.

About Spinal Muscular Atrophy
Spinal muscular atrophy (SMA) is an autosomal-recessive genetic disorder characterized by lower motor neuron loss and progressive muscle weakness. SMA is caused by a genetic defect in the SMN1 gene which encodes for SMN, a protein necessary for survival of motor neurons. SMA kills more infants than any other genetic disease in today's world.

About AveXis, Inc.
Based in Dallas, Texas, AveXis is a clinic-stage gene therapy platform company establishing unique industry alliances to create innovative treatments for people with unmet medical needs. Spinal muscular atrophy (SMA) is the company’s first focus. For more information regarding AveXis, please visit www.avexisinc.com.
AveXis Inc.
Jillian Bowman