An experimental gene therapy based on RNA interference (RNAi) shows potential to treat patients with familial amyotrophic lateral sclerosis (ALS) caused by mutations in the superoxide dismutase 1 (SOD1) gene, according to results of a preclinical study in nonhuman primates. The study, “Safe and effective superoxide…
Cytokinetics has completed patient enrollment in its Phase 2 trial to evaluate the safety and potential of reldesemtiv to improve muscle function in patients with amyotrophic lateral sclerosis (ALS), the company announced. The trial (NCT03160898), called FORTITUDE-ALS, includes about 450 ALS patients in the U.S., Canada, Europe,…
The amount of time it takes to diagnose amyotrophic lateral sclerosis (ALS) and the use of Rilutek (riluzole) to treat it vary significantly across Canada, according to a new nationwide analysis of a patient registry. These findings highlight the need for future studies to identify the factors that contribute…
About four years ago, ALS impeded my speaking ability. Like more than 80 percent of all ALS patients, I now have dysarthria, or what I refer to as my “ALS voice.” It’s a slurred, slow speech pattern with a nasal tone and imprecise pronunciation of consonants. For…
The U.S. Food and Drug Administration (FDA) has accepted for review the new drug application (NDA) of Biohaven Pharmaceuticals’ BHV-0223, a sublingual (dissolved under the tongue) form of riluzole, as a treatment for amyotrophic lateral sclerosis (ALS). Biohaven has begun enrollment of ALS patients for its Phase 2/3…
The brain’s waste clearance pathway, known as the glymphatic system, could enable more efficient delivery of therapeutics into the brain for the treatment of disorders such as amyotrophic lateral sclerosis (ALS), Alzheimer’s, Parkinson’s, and brain cancer, according to a study. The study, “Transcranial optical…
Amyotrophic lateral sclerosis (ALS) or frontotemporal lobar degeneration (FTLD) patients with the C9ORF72 mutation are at an unusually high risk of malignant skin cancer and should be monitored for it, a study suggests. The study, “Increased risk of melanoma in C9ORF72…
Before ALS, whenever I thought about the future, I assumed my life would continue in a state of health and happiness, filled with lots of interesting activities. But in 2010, when I learned I had ALS, thinking about my future became very dismal and frightening. My mind…
Loss of C9orf72, a protein whose corresponding gene is among those most often mutated in amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD), causes an imbalance in the metabolism of fats (lipids) in motor neurons, a new study shows. These findings suggest that an imbalance in energy and lipid metabolism…
Health conditions that may lead to a faster metabolism, or higher metabolic rate, may contribute to the development of amyotrophic lateral sclerosis (ALS), a large-scale, population-based study suggests. The study, “Finding diseases associated with amyotrophic lateral sclerosis: a total population-based case–control study,” was published in the…
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