“It is a rare disease like many of the diseases that we deal with by definition, but within that context it is really one of the most common. In fact, it’s the second most common autosomal recessive disease that we deal with, the most common being cystic fibrosis and such.”
Research is key to improving life expectancy and quality of life for patients. Progress reports on six treatment options for SMA, all currently in clinical trials, were spotlighted recently at the 2016 Annual SMA Conference in Anaheim, CA.
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