Mutations in the FUS gene, which cause some forms of amyotrophic lateral sclerosis (ALS), result in a circular RNA molecule called circ-Hdgfrp3 being pulled into toxic protein clumps that form in nerve cells, according to a study. This is the first time that a circular RNA has been shown…
The Cullen Education and Research Fund (CERF), a private philanthropy based in London, has awarded more than $800,000 to support two U.S. research projects that are working to improve life for people with amyotrophic lateral sclerosis (ALS). The CERF Medical Engineering Prize, worth €500,000 (about $570,000), was awarded to…
NP001 slowed functional and lung declines in a subset of middle-aged patients with amyotrophic lateral sclerosis (ALS) who had high levels of inflammation, according to an analysis of previous data from Phase 2a and 2b clinical trials. While earlier results failed to show overall…
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…
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