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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…

Clusterin, a protein that plays a key role in motor neuron health, and ficolin-3, a protein essential for the activation of immune responses, may be promising biomarkers of amyotrophic lateral sclerosis (ALS), according to a recent study. The study, “Levels of clusterin, CD5L, ficolin-3,…

The EveryLife Foundation for Rare Diseases has launched a nationwide National Burden of Rare Disease Survey to measure the full implications, economic and social, of living with rare disease in the United States. People with rare diseases know that the impacts of such conditions extend beyond just medical…

Long delays between symptom onset and diagnosis are common with amyotrophic lateral sclerosis (ALS), and need not be if general practitioners (GPs) were more aware of the disease and its likely “red flags,” a study suggests. Its researchers highlighted specific “flags,” like difficulties with speech or swallowing or muscle…