The latest issue of Compass, our research publication, is now available online.
This Fall 2014 issue includes news from our 2014 SMA researcher meeting, and news from the 2014 CME conference.
Also in this issue, Cure SMA Scientific Advisory Board member Arthur Burghes, PhD, provides an update from our 2014 conference special session, “Moving Beyond SMN? Strategies to Identify Non-SMN Drug Targets for SMA.”
SMA is caused by a mutation in the survival motor neuron gene 1 (SMN1). In a healthy person, this gene produces a protein—called survival motor neuron protein or SMN protein—that is critical to the function of the nerves that control our muscles. Individuals with SMA do not produce this protein at high enough levels.
Scientists are pursuing many treatment strategies that address the loss of SMN protein. But other treatment strategies may also be effective. This special session looked at some of these other treatment strategies, including what we can learn from how other diseases are being treated.
You can download this latest issue from our research publications page, where you can also browse other past issues of Compass.