A new mutation in the FUS gene, which results in a shorter version of the FUS protein that cannot enter the cell nucleus to work as it should, may cause an aggressive from of juvenile amyotrophic lateral sclerosis (ALS), the case of an adolescent girl shows. The…
A single dose of an artificial microRNA — a tiny RNA molecule that is able to control the activity of certain genes — can lower the activity of SOD1, a gene commonly mutated in patients with familial amyotrophic lateral sclerosis (ALS), a proof-of-concept study shows. According to researchers,…
Changes in commonly tested blood biomarkers in people with amyotrophic lateral sclerosis (ALS) may be associated with faster disease progression and an increased risk of mortality, according to a recent study. Given the easy access and low cost of these standard…
Tofersen, Biogen’s investigational therapy for people with familial amyotrophic lateral sclerosis (ALS) caused by SOD1 gene mutations, was safe and generally well-tolerated over three months, and appeared to lower SOD1 protein levels in the central nervous system, a Phase 1/2 clinical trial has found. There…
Bereaved caregivers of people with motor neurone disease (MND) are at increased risk of prolonged grief disorder compared to the general bereaved population, a new study suggests. This indicates a need for greater support for bereaved caregivers of people with MND. The study, “Grief, depression, and anxiety…
A comparison of amyotrophic lateral sclerosis (ALS) among U.S. military veterans found those who lived the longest with this disease — an average of 16.3 years in more than 40% of those studied — had a younger age at onset but slower initial progression. Findings…
Low doses of Clinigen’s aldesleukin, an immunotherapy used in certain types of cancer, safely boosted the number and function of regulatory T-cells (Tregs), a type of immune cell that keeps others in check, in people with amyotrophic lateral sclerosis (ALS), according to data from a pivotal Phase 2…
The HudsonAlpha Institute for Biotechnology has received a $20,000 ALS Association grant to continue its research collaboration with Crestwood Medical Center in Alabama. Called “Impacting ALS,” the project’s goal is to identify genetic changes that contribute to amyotrophic lateral sclerosis (ALS), with the overarching goal…
microRNAs found in brain-derived exosomes — microscopic vesicles containing genetic material, proteins and fats shed by cells into the bloodstream — may be useful blood biomarkers to diagnose amyotrophic lateral sclerosis (ALS), a study reported.
The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to Clinigen’s aldesleukin for the treatment of amyotrophic lateral sclerosis (ALS). Aldesleukin is a lab-made version of the interleukin 2 (IL-2), an immune signalling molecule known to play a key role in the maintenance of a…
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