– Includes Results from Phase 1 Trial of AVXS-101 in SMA Type 1 –
– Conference call and webcast Tuesday, April 25 at 4:30 p.m. EDT –
CHICAGO, April 18, 2017 -- AveXis, Inc. (NASDAQ:AVXS), a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, will host a live webcast briefing of AVXS-101 data as presented at the 2017 Annual Meeting of the American Academy of Neurology in Boston, including results from the Phase 1 trial of AVXS-101 in spinal muscular atrophy (SMA) Type 1. Details of these five preclinical and clinical abstracts can be found in a press release issued on March 2, 2017, available on the Investors and Media section of the AveXis website.
The webcast will take place Tuesday, April 25, 2017 at 4:30 p.m. EDT. Analysts and investors can participate in the conference call by dialing (844) 889-6863 for domestic callers and (661) 378-9762 for international callers, using the conference ID 10345273. The webcast can be accessed live on the Events and Presentations page in the Investors and Media section of the AveXis website, www.AveXis.com. The webcast will be archived on the company’s website for 5 days and will be available for telephonic replay for 14 days following the call by dialing (855) 859-2056 (Domestic) or (404) 537-3406 (International), conference ID 10345273.
About SMA
SMA is a severe neuromuscular disease characterized by the loss of motor neurons leading to progressive muscle weakness and paralysis. SMA is caused by a genetic defect in the SMN1 gene that codes SMN, a protein necessary for survival of motor neurons. The incidence of SMA is approximately one in 10,000 live births. SMA is the leading genetic cause of infant mortality.
The most severe form of SMA is Type 1, a lethal genetic disorder characterized by motor neuron loss and associated muscle deterioration, which results in mortality or the need for permanent ventilation support before the age of two for greater than 90 percent of patients.
About AVXS-101
AVXS-101 is a proprietary gene therapy candidate of a one-time treatment for SMA Type 1 and is designed to address the monogenic root cause of SMA and prevent further muscle degeneration by addressing the defective and/or loss of the primary SMN1 gene. AVXS-101 also targets motor neurons providing rapid onset of effect and crosses the blood brain barrier allowing an IV dosing route and effective targeting of both central and systemic features.
About AveXis, Inc.
AveXis is a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases. The company’s initial proprietary gene therapy candidate, AVXS-101, recently completed a Phase 1 clinical trial for the treatment of SMA Type 1. For additional information, please visit www.avexis.com.
Media Inquiries: Lauren Barbiero W2O Group 646-564-2156 [email protected] Investor Inquiries: Jim Goff AveXis, Inc. 650-862-4134 [email protected]


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