News

An abundance of events are afoot around the world to mark Rare Disease Day 2020 on Feb. 29. The activities are focused on heightening awareness about rare diseases and the hundreds of millions of individuals they are thought to affect. Patients, caregivers, and advocates worldwide will sport denim ribbons…

The protein synaptotagmin 13 (SYT13) protected motor neurons from degeneration in cell and animal models of amyotrophic lateral sclerosis (ALS) and spinal muscular atrophy (SMA), a study reports, suggesting a gene therapy based on this protein’s related gene, SYT13, would treat these and other diseases of motor nerve cells.

Impaired processing and buildup of protein aggregates associated with amyotrophic lateral sclerosis (ALS) are linked to a protein that regulates the uptake of substances into the cell, a process known as endocytosis, a study found. Enhancing endocytosis may be an…

People with amyotrophic lateral sclerosis (ALS) may benefit from at-home monitoring using an app, a study found. The study, “Telehealth as part of specialized ALS care: feasibility and user experiences with ‘ALS home-monitoring and coaching’,” was published in Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration. Providing…

Antibodies that target proteins wrongly expressed in the brain and driven by mutations in C9ORF72, a cause of amyotrophic lateral sclerosis (ALS), eased symptoms and prolonged survival in a mouse disease model, a new study shows. Its researchers suggest that such antibodies could be used to treat people with…

Cognitive impairment in amyotrophic lateral sclerosis (ALS) may be linked to the buildup of the protein TDP-43 in the brain, a new study suggests. However, TDP-43 alone likely isn’t the cause of such impairment, and due to the study’s small sample size, more research is needed to clarify…

The biotechnological company INmune Bio has been awarded a $500,000 grant from the ALS Association to further develop a therapy that might reprogram the innate immune system in people with amyotrophic lateral sclerosis…

PrimeC, a new combination treatment by NeuroSense Therapeutics to slow down or halt the progression of amyotrophic lateral sclerosis (ALS), has received orphan drug status from the U.S. Food and Drug Administration (FDA), and is being investigated in two recently-initiated clinical trials. The decision to…

The U.S. Food and Drug Administration (FDA), a vast government bureaucracy, employs about 17,500 people and had a budget of $5.7 billion in 2019. Yet even with its enormous resources, the FDA these days relies more and more on patients to…

A new spinal cell delivery method may reduce the risks and boost the effectiveness of stem cell therapy designed to regenerate the nervous system in neurodegenerative diseases such as amyotrophic lateral sclerosis (ALS), an early study in rats has found. The study, “Spinal parenchymal occupation by…