Shares of AveXis Inc. (AVXS) have gained more than 50% year-to-date and trade around $74.00.
AveXis' investigational treatment for spinal muscular atrophy is AVXS-101, a gene therapy designed to replace the defective or lost SMN gene. As you may know, mutations in the human survival motor neuron 1 (SMN) gene are the primary cause of spinal muscular atrophy.
Spinal Muscular Atrophy, or SMA, is the number 1 genetic cause of infant death. Based on the age of the initial onset of muscle weakness and severity of the disease, spinal muscular atrophy is classified into four types: Type I (Infantile), Type II (Intermediate), Type III (Juvenile) and Type IV (Adult onset).
The first drug approved to treat children and adults with spinal muscular atrophy is Spinraza, developed by Ionis and Biogen. It was approved by FDA last December.
To know which other companies are developing a treatment for SMA, read our articleThe Race To Cure Spinal Muscular Atrophy.
AVXS-101 is currently in an ongoing phase I clinical trial for the treatment of SMA Type 1, the most severe form of SMA. The top line results from this study are expected to be reported today.
Will the results cheer investors or disappoint them?
We alerted our subscribers to AVXS on September 15, 2016 when it was trading around $38. The stock has been on the rise since then, and it touched an all-time intraday high of $75.48 yesterday before closing the day's trading at $74.88.
Do you want to hear how AVXS-101 fared in the phase I trial? Be all ears...
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by RTT Staff Writer
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