Edaravone — now more widely known by its U.S. brand name, Radicava — has produced “a minor miracle” for some amyotrophic lateral sclerosis patients, said Stephen Byer, co-director of ALS Worldwide. “We have found and continue to find great benefit in terms of slowing, stopping and, in some…
News
University of Michigan and University of Pennsylvania researchers used a cutting-edge technique called cryo-electron microscopy to see how the Hsp104 yeast enzyme breaks up clumps of the misfolded proteins that are hallmarks of neurodegenerative diseases like ALS. The resolution that the technique provided was so high that it was close to the level…
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…
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