Researchers have long sought to explain precisely how the most common genetic mutation linked to both amyotrophic lateral sclerosis (ALS) and frontotemporal dementia causes the death of nerve cells. Now, Johns Hopkins Medicine researchers report new evidence that the mutation slowly disrupts the vital transport system of proteins, enzymes and other material in and out […]
Investigational ALS drug generates promising clinical trial results
An experimental medication slows the progression of the neurodegenerative disease called Amyotrophic lateral sclerosis (ALS), or Lou Gehrig’s disease, according to recently released results from a clinical trial run by investigators at the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital (MGH) and Amylyx Pharmaceuticals, Inc., the company that manufactures the […]
Gene therapy can protect against ALS and SMA-related cell death
Researchers at Karolinska Institutet in Sweden and the University of Milan in Italy have identified a gene in human neurons that protects against the degeneration of motor neurons in the deadly diseases ALS and SMA. Gene therapy in animal models of these diseases was shown to protect against cell death and increase life expectancy. The […]