News

Since 2008, Rare Disease Day — the last day of February — has brought together patients, caregivers, family members, friends, and advocates from around the world to raise awareness and improve equity for the more than 7,000 known rare diseases that affect more than 300 million people. In 2022, the…

An experimental antisense oligonucleotide that works to suppress the mutant C9orf72 gene — a cause of amyotrophic lateral sclerosis (ALS) — safely lowered the production of damaging proteins and other molecules in a patient in a pilot trial. “While other teams have documented that this gene can be suppressed in cells…

A mouse model of amyotrophic lateral sclerosis (ALS) carrying a mutation in the SOD1 gene showed alterations in the gut microbiome, followed by motor impairments and defects in enteric nervous system — the gut’s own autonomous nervous system — compared with healthy mice, a study found. These microbiome changes occurred…

The Canadian Neuromuscular Disease Registry (CNDR) and Amylyx Pharmaceuticals are collaborating on an initiative that could produce the first real-world evidence on AMX0035, Amylyx’s investigational therapy to slow functional decline in amyotrophic lateral sclerosis (ALS) patients. The two, in partnership with neuromuscular centers across Canada, will collect…

More than half of the people with amyotrophic lateral sclerosis (ALS) responding to a survey were dissatisfied with their quality of life and pessimistic about their future. The U.S.-based survey was conducted by ALS News Today. Nevertheless, the vast majority of respondents reported to be satisfied with their…

A team of scientists in the U.K. and Japan has determined the structure of aggregated TDP-43, the protein whose abnormal clumps are characteristic of amyotrophic lateral sclerosis (ALS). Their work, reportedly the first to reveal the molecular structure of aggregated TDP-43, identified a “double-spiral fold” of the protein in patients’ brain…

To help support people living with motor neurone disease (MND), a group of disorders that includes amyotrophic lateral sclerosis, the My Name’5 Doddie Foundation has donated £100,000 ($135,000) to the MND Association. The association will use the funds to bolster its financial support grant program, which…

Cytokinetics has awarded a total of $100,000 to five nonprofit patient advocacy organizations to support efforts to enhance communications, increase disease awareness, and boost community engagement. Recipients of the fourth annual Communications Fellowship Grant Program include the Les Turner ALS Foundation, the Northern Ohio Chapter of The…

Wave Life Sciences has gained additional financial support for an ongoing “basket” Phase 1b/2a clinical trial evaluating its experimental therapy, WVE-004, in people with amyotrophic lateral sclerosis (ALS) and/or frontotemporal dementia (FTD) caused by mutations in the C9orf72 gene. So-called basket trials, commonly used in the field of cancer, test…

Eikonoklastes Therapeutics has added a candidate gene therapy for amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases to its pipeline. The announcement follows the completion of a licensing agreement with the University of California San Diego. Brian Head, PhD, a professor with the university’s department of anesthesiology, and…