Isis Pharmaceuticals

Normal SOD1 protein, like mutant SOD1 protein, appears to be prone to misfolding and can cause ALS, at least in mice

Posted on Friday, October 26, 2012 - 05:00, By: Amy Madsen
It's been known since the early 1990s that mutations in the superoxide dismutase-1 (SOD1) gene can result in the production of any number of varieties of improperly folded (misfolded) SOD1 protein, and that these misfolded proteins can cause familial amyotrophic lateral sclerosis (ALS).

Human testing has begun of ISIS-SOD1-Rx, a compound designed to block production of a toxic protein in people with the SOD1-related form of familial (inherited) ALS

Posted on Friday, March 5, 2010 - 15:20, By: Amy Madsen
Isis Pharmaceuticals of Carlsbad, Calif., has begun a phase 1 clinical trial of its experimental compound ISIS-SOD1-Rx in people with familial (inherited) ALS caused by toxic SOD1 protein molecules.