Familial ALS

A subtype of astrocytes — cells that normally protect motor neurons — causes 'unprecedented toxicity' to motor neurons in a rat model of familial ALS

Posted on Thursday, October 27, 2011 - 05:00, By: Amy Madsen
A multinational team of scientists has identified a specific type of astrocyte that behaves abnormally, causing degeneration and death in motor neurons in rats with a disease resembling familial SOD1-related ALS (amyotrophic lateral sclerosis, or Lou Gehrig's disease). The astrocyctes have been...

Small molecules in the spinal cords of symptom-free people with ALS-associated SOD1 mutations appear to signal the presence of disease before symptom onset

Posted on Thursday, October 13, 2011 - 16:00, By: Amy Madsen
An MDA-supported team of scientists has identified specific changes that occur in the spinal cord prior to disease onset in people with familial (inherited) ALS related to mutations in the SOD1 gene.

The registry, called fALS Connect, is for people with familial ALS and has the goal of hastening research progress in this disease

Posted on Friday, October 7, 2011 - 06:00, By: Amy Madsen
A new registry has been launched for people with the familial (inherited) form of amyotrophic lateral sclerosis (ALS). The Web-based fALS Connect registry was developed by a research team from the University of Miami Miller School of Medicine in Florida. The team aims to connect families affected...

Early results show ISIS-SOD1-Rx, an antisense drug directed against the SOD1 form of familial ALS, appears safe in the lowest-dose group

Posted on Wednesday, April 27, 2011 - 16:12, By: Margaret Wahl
  Update (July 21, 2014): This study was completed in 2012. ========================================================================== original story:

Isis Pharmaceuticals hosted a webinar March 2, 2011, to explain its phase 1 trial for people with SOD1-related familial ALS

Posted on Wednesday, February 2, 2011 - 13:16, By: Margaret Wahl
Isis Pharmaceuticals hosted an interactive webinar Wednesday, March 2, 2011, at 12 noon Eastern Standard Time, to help prospective participants understand its MDA-supported, phase 1 study of ISIS-SOD1-Rx for people with familial amyotrophic lateral sclerosis (ALS) due to mutations in the SOD1 gene...

Researchers have found that some ALS-related TDP43 mutations cause the TDP43 protein to hang around too long, which can be toxic to cells

Posted on Wednesday, August 4, 2010 - 11:14, By: Margaret Wahl
At least some of the mutations in the TDP43 protein that are known to lead to ALS cause the TDP43 protein to be more stable than usual and change its interactions with other cellular proteins, say researchers at the University of California-San Diego. The changes are toxic ones.

ALS TDI webinar describes progress in familial ALS research

Posted on Saturday, May 1, 2010 - 13:25, By: Amy Madsen
Although familial, or inherited, ALS (FALS) and the sporadic (uninherited) form of the disease demonstrate similar progression patterns after symptoms appear, the causes for sporadic ALS remain elusive, while a number of genetic mutations that cause FALS have been — and continue to be — uncovered.