SMA therapy

A mouse model of adult-onset spinal muscular atrophy, developed with MDA support, suggests a treatment now in testing for early-onset disease may benefit adults

posted on September 17, 2013 - 2:25pm
Scientists supported in part by MDA have developed a new research mouse model with a disorder that mimics the adult-onset form of chromosome 5-related spinal muscular atrophy (SMA), a genetic disease of spinal nerve cells that control muscle activity (motor neurons).  

Zinc finger protein 1 (ZPR1) appears to be a 'protective modifier' of spinal muscular atrophy, making it a potential target in the development of therapies

posted on March 27, 2012 - 10:44am
Adequate levels of zinc finger protein 1 (ZPR1) appear to be a "protective" modifier of spinal muscular atrophy (SMA), an MDA-supported team of scientists has reported. Modifiers influence disease onset and severity by changing various biological pathways. The identification of ZPR1 as a modifier reveals a potential target for therapy development and also sheds light on the mechanisms that drive...