News

People with mutations in the gene coding for tumor protein p73 (TP73) are at higher risk of developing amyotrophic lateral sclerosis (ALS), researchers report. Their study, “Loss of TP73 function contributes to amyotrophic lateral sclerosis pathogenesis,” was published online in bioRxiv. There’s no doubt that some genes…

About 100 scientists, researchers, pharmaceutical executives, and others will converge on Austria’s capital city early next month for the 2nd International Congress on Advanced Treatments in Rare Diseases. The March 4-5 meeting, to take place at the Hilton Am Stadtpark Vienna, features 27 speakers on a variety of disorders…

Enhanced production of a protein called Vps4 was shown to delay the degenerative process in injured nerve cells, which may help researchers to better understand and treat nerve damage in neurodegenerative disorders such as Parkinson’s disease and amyotrophic lateral sclerosis (ALS), a study says. The findings were published…

A gene therapy candidate targeting a key amyotrophic lateral sclerosis (ALS) mutation in the C9orf72 gene is able to lower the accumulation of toxic RNA clumps and reduce the activity of this mutated gene, in cells collected from a patient with frontotemporal dementia (FTD) and in a…

High LDL cholesterol levels, also known as “bad cholesterol,” may contribute to the development of amyotrophic lateral sclerosis (ALS), researchers report. Their study, “Shared polygenic risk and causal inferences in amyotrophic lateral sclerosis” was published in Annals of Neurology. Several lifestyle and other factors are thought to…

The ALS Association’s new #VoiceYourLove awareness campaign is getting a boost from celebrities and the TRON Foundation, a leading blockchain protocol. The initiative seeks to honor ALS patients who have lost the ability to speak, with the ultimate goal of discovering new therapies and a cure for…

Scores of events are afoot worldwide to mark Feb. 28, Rare Disease Day 2019. The activities aim to raise awareness about rare diseases and the millions of people — estimates run as high as 350 million — they are thought to impact. Across countries, patients, caregivers and advocates will paint faces, wear…

Using a recumbent stepper is a safe and well-tolerated way for patients with amyotrophic lateral sclerosis (ALS) to do physical exercise, a pilot study suggests. The study, “Recumbent stepping aerobic exercise in amyotrophic lateral sclerosis: a pilot study,” was published…

GPM Investments raised $244,263 for patients with amyotrophic lateral sclerosis (ALS) and other neuromuscular disorders in its eighth holiday pinup fundraiser in 2018 in partnership the Muscular Dystrophy Association. Approximately 1,400 GPM convenience stores throughout the East Coast and Midwest regions of the U.S. participated in the campaign, including…

A potential therapy for amyotrophic lateral sclerosis (ALS) consisting of an antibody that reduces the abnormal accumulation of the TDP-43 protein improved cognitive and motor performance in mice, a study shows. The study, “Viral-mediated delivery of antibody targeting TAR DNA–binding protein 43 mitigates associated neuropathology,” was published in The…