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of acute reversible illness or perioperatively, as observed in the Finkel 2014 study, along with major developmental milestones for a healthy baby.
Existing Treatments for SMA
At this time, there is no FDA-approved treatment for SMA. The current standard of care for patients with SMA is
limited to palliative therapies, including life-long respiratory care, ventilator support, nutritional care, orthopedic care and physical therapy. Respiratory care is critical for patients with SMA
Type 1 and Type 2, as the cause of death in these subpopulations is usually respiratory-related.
Our Product Candidate: AVXS-101 for the Treatment of SMA Type 1
AVXS-101 is our proprietary gene therapy product candidate for the treatment of SMA Type 1. Because SMA is a
neurodegenerative disease, reduced levels of SMN protein lead to continued degeneration. The goal of AVXS-101 is to give patients a one-time treatment to restore the body's production of SMN protein
to prevent further degeneration. Based on preliminary observations of our ongoing Phase 1 clinical trial, we believe that AVXS-101 also enables increased motor function. AVXS-101 contains the
four elements that we believe are necessary for optimal delivery and function.
Components of AVXS-101