Clinical Trials and Studies

Results of a large-scale trial of tirasemtiv in ALS show the drug may slow respiratory decline without improving scores on the ALS Functional Rating Scale

Posted on Thursday, May 1, 2014 - 14:07, By: Margaret Wahl
The experimental drug tirasemtiv, in development by South San Francisco biopharmaceutical company Cyokinetics as a potential treatment for amyotrophic lateral sclerosis (ALS), slowed the decline in at least one measure of respiratory function in a recent, large-scale study known as BENEFIT-ALS (...

An MDA-supported research team has found that expansions of the C9ORF72 gene cause formation of abnormally shaped DNA and RNA, with adverse effects on cells

Posted on Wednesday, April 23, 2014 - 15:11, By: Margaret Wahl
Jiou Wang, an MDA research grantee at Johns Hopkins University, says the new findings could have implications for several disorders.

Findings show TDP43 protein transports RNA molecules in motor neurons; impairment of the function may help drive the amyotrophic lateral sclerosis disease process

Posted on Wednesday, March 12, 2014 - 14:28, By: Amy Madsen
TDP43 protein is involved in the transport of vital cargo within motor neurons, the muscle-controlling nerve cells that are lost in amyotrophic lateral sclerosis (ALS), a multinational research team reports. Further, disruption of the function results from ALS-causing mutations in the gene for the...

An MDA-supported study of 24 ALS patients showed those on high-calorie, high-carbohydrate tube feedings fared better than those on control or high-calorie, high-fat formulas

Posted on Wednesday, March 5, 2014 - 15:19, By: Margaret Wahl
A study supported in part by MDA has found that a high-calorie, high-carbohydrate diet is safe and well-tolerated in late-stage amyotrophic lateral sclerosis (ALS), that it leads to moderate weight gain, and that it may slow disease progression.

Researchers say the new model will increase understanding of the role FUS mutations play in ALS and help pinpoint therapeutic targets for FUS-associated ALS

Posted on Monday, February 10, 2014 - 11:45, By: Amy Madsen
A new research mouse model is the first to carry a mutation in the gene for the FUS protein that's known to cause human amyotrophic lateral sclerosis (ALS), an MDA-supported research team reports. Animal models play a key role in disease research, and researchers say the new FUS model is an...

An MDA-supported team of researchers has shown that 'reprogrammed' versions of the yeast protein HSP104 can reverse protein misfolding and clumping in amyotrophic lateral sclerosis  

Posted on Monday, January 27, 2014 - 09:49, By: Amy Madsen
In studies conducted in yeast cells, modified versions of a common yeast protein broke up aggregates (clumps) of improperly folded proteins known to be involved in amyotrophic lateral sclerosis (ALS) and helped the misfolded proteins re-fold into their normal shape, an MDA-supported team of...

Themes discussed at a recent symposium included immune system modulation, combatting toxic proteins and genetic material, replacing or repairing damaged cells, and improving muscle function

Posted on Friday, December 20, 2013 - 10:25, By: Margaret Wahl
"We are undoubtedly slowing down the MND [motor neuron disease] supertanker, and can start to see how we might turn it around for the first tim," said Martin Turner, senior clinician scientist at the University of Oxford (United Kingdom), on research being reported on at the 24th International...