News

Harvard Medical School researchers have discovered how a mutation of the C9ORF72 gene causes the changes in energy production and nerve cell function that are linked to inherited forms of ALS and frontotemporal dementia, or FTD. The mutation produces two toxic proteins that can disrupt the normal processing of genetic instructions,…

Measuring neurofilaments in brain and spinal fluid appears to be a reliable way of confirming an ALS diagnosis, Belgian and German researchers report. This finding is important because it takes an average of a year from the appearance of the first symptoms before doctors can diagnose the disease. The discovery could…

The Israel Innovation Authority (IIA) has awarded BrainStorm Cell Therapeutics a $2.1 million grant to support the development of NurOwn, an innovative mesenchymal stem cell-based platform to treat neurodegenerative diseases including amyotrophic lateral sclerosis (ALS).

Several metals may be linked to the risk of amyotrophic lateral sclerosis (ALS), according to results of a small study in Italy. The findings state that levels of metals and other essential trace elements in blood, urine, and hair may serve as biomarkers to diagnose and monitor ALS. The study titled,…

The former malaria therapy pyrimethamine reduces levels of an ALS-linked protein in the spinal fluid of patients whose disease stems from gene mutations, a Phase 1/2 clinical trial shows. The protein is SOD1. The mutations connected with amyotrophic lateral sclerosis are in the SOD1 gene that controls the protein. The…

Mallinckrodt has launched the Phase 2b PENNANT trial (NCT03068754) to investigate the safety and effectiveness of H.P. Acthar Gel (repository corticotropin injection) as a treatment for amyotrophic lateral sclerosis (ALS). The H.P. Acthar Gel is an experimental injectable drug that the U.S. Food and Drug Administration (FDA) has granted fast…

The U.S. Food and Drug Administration has expanded its approval of Dysport (abobotulinumtoxinA) as a  treatment for spasticity in adults from not just the upper limbs but to the lower limbs as well, according to its maker, Ipsen Biopharmaceuticals. Spasticity can affect those with amyotrophic lateral sclerosis (ALS), multiple sclerosis, cerebral…

Stem cell transplantation in patients with amyotrophic lateral sclerosis (ALS) has the potential to be “an important alternative strategy” in treating the disease, a new study suggests. In recent decades, many advances in identifying and understanding the underlying mechanisms of ALS have been made. But those advances have not resulted in…

A new study shows that patients with amyotrophic lateral sclerosis (ALS) experience decreased oxidative metabolism during exercise, and that differences among individuals in exercise capacity matches ALS clinical heterogeneity. The study, titled, “Inefficient skeletal muscle oxidative function…

AveXis and REGENXBIO have signed a new licensing agreement to develop therapies for a particular form of ALS and for Rett syndrome. They already have an agreement that covers spinal muscular atrophy therapies. The form of amyotrophic lateral sclerosis that the new agreement covers is the one caused by mutations…