Early results of a phase 1 clinical trial of ISIS-SOD1-Rx, an antisense oligonucleotide drug for SOD1-related amyotrophic lateral sclerosis (ALS), show the drug appears to be safe and well tolerated at the lowest dosage level, based on a group of six patients who received 12-hour infusions of it into their spinal fluid.
Most people affected by neuromuscular disease know all too well that their condition is costly. MDA wants to find out just how costly — and then translate those figures into terms that will speak to policy makers.
(Update 9/14/11: The 2011 scholarship winners have been selected. To view a list of winners visit www.deshae.org/cmms/awards/2011/scholars.pdf. Applications for quality of life grants continue to be accepted and are awarded on an ongoing basis.)
Scientists have found that the protein TDP43 normally influences the synthesis of more than 1,500 other proteins, including itself. Mutations in the gene for TDP43 are a known cause of ALS, and TDP43 is depleted in the nucleus and enriched in the cytoplasm (main cell compartment) in ALS-affected nerve cells, even when no TDP43 mutations exist. Understanding TDP43's many functions is likely to improve understanding of ALS.