Toxic RNA buildup appears to underlie nerve cell death in C9ORF72 amyotrophic lateral sclerosis and frontotemporal dementia; antisense therapies may help  

Posted on Tuesday, November 19, 2013 - 05:00, By: Amy Madsen
Results from three recent studies — two supported in part by MDA — shed light on how the chromosome 9 open reading frame 72 (C9ORF72) mutation causes nerve cell death in amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD).

Biomarkers are needed to light the way in research and therapy development

Posted on Sunday, July 1, 2012 - 09:45, By: Other
An urgent need exists for biomarkers — biological indicators — in ALS research that can be used:
Posted on Sunday, July 1, 2012 - 09:41, By: Other
Bryan Traynor Even as some scientists work to develop animal and human cellular models of C9ORF72-related ALS (see C9ORF72 Research Models), others already are targeting the gene with experimental therapies designed to inactivate it or the repeat expansion it harbors.

Posted on Saturday, January 1, 2011 - 13:21, By: Margaret Wahl
“It never made sense to me that ALS spreads,” says Marc Diamond, an associate professor of neurology at Washington University in St. Louis. “Why don’t just a few cells die, and that’s it?” That’s a question that has bothered Diamond, who now has an MDA grant to study the subject.

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.