Experimental Gene Therapy Successfully Silences Key ALS Gene C9orf72, Preclinical Studies Show

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…

“Brute Force and Ignorance” That is the title of a song, written by the late Irish bluesman Rory Gallagher. It also is a fair depiction of the human, knee-jerk reaction to a seemingly unsolvable problem, such as banging on the television to address erratic reception or kicking the…

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…

Last week was an exciting one for me: The website I created went live. Working on this special project was an entirely new experience; it turned out to be a challenging one, but from beginning to end it gave me a sense of purpose and value. Why did…

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…

Warning the reporter accompanying him not to take any pictures, veteran horticulturalist Michael Castleman punches an electronic code and unlocks the door to Room 209, nicknamed the “Mother Room.” Photography is indeed forbidden inside this living vault, which contains 20 phenotypes of cannabis plants thriving under the glare of 25…