SOD1

A diminished ability to regulate body temperature may be a manifestation of amyotrophic lateral sclerosis (ALS), results of a mice study suggest. Such a diminishment could be related to problems in controlling the naturally occurring circadian clocks that regulate tissues and organs. The circadian rhythm is an important mechanism that…

IONIS-SOD1Rx (BIIB067), an investigational therapy for amyotrophic lateral sclerosis (ALS) developed by Biogen and Ionis Pharmaceuticals, was shown to prolong survival and reverse neuromuscular damage in mouse and rat models…

Gene editing may be a viable tool to slow disease-causing processes in amyotrophic lateral sclerosis (ALS) caused by mutations in the SOD1 gene, a mouse study indicates. Published in the journal Science Advances, the study demonstrated that the method delayed disease…

Voyager Therapeutics has recently presented data of its leading adeno-associated virus (AAV) gene therapy for several neurological diseases, including amyotrophic lateral sclerosis (ALS), at the Congress of the European Society of Gene and Cell Therapy (ESGCT), Oct. 17-20 in Berlin, Germany. In their poster, “Translation of Intrathecal Delivery of an AAV Gene…

Genervon Biopharmaceuticals has published the results of a somewhat promising Phase 2a clinical trial investigating the effects of GM604 (or GM6) in patients with amyotrophic lateral sclerosis (ALS). The study, “A Phase 2A randomized, double-blind, placebo-controlled pilot trial of GM604 in patients with Amyotrophic Lateral Sclerosis (ALS Protocol GALS-001)…

Genervon Biopharmaceuticals has published a list of 89 genes associated with amyotrophic lateral sclerosis (ALS) that are modulated by its drug candidate GM6. The identification of the genes regulated by GM6 helps to explain the mechanism of action of this treatment, which aims to improve neuronal repair and survival, and…

The biopharma Neurimmune and the venture capital fund TVM Life Science Ventures VII announced they have created a Swiss company called AL-S Pharma AG to develop a new therapy for the treatment of amyotrophic lateral sclerosis (ALS). AL-S Pharma AG is a special purpose company formed to develop one compound — AP-101…

Researchers at Nagoya University in Japan, in collaboration with several U.S. institutions, suggest that the collapse of the mitochondria‐associated membranes (MAM) is linked to the development of amyotrophic lateral sclerosis (ALS). Mitochondria‐associated membranes regulate calcium levels, mitochondrial function, and cell death in the body, and have been linked to neurodegenerative…

Researchers have identified a factor that prevents SOD1 — a protein causing amyotrophic lateral sclerosis (ALS) in a proportion of patients — from misfolding. The finding may advance the development of new drug therapies to stop or slow progression of the disease. The study, “Endogenous macrophage migration inhibitory factor reduces the…

By introducing a mutation in SOD1 — the protein believed to be responsible for a large part of amyotrophic lateral sclerosis (ALS) cases — researchers managed to prevent the protein from clumping in a way that kills neurons. The study, “A Phosphomimetic Mutation Stabilizes SOD1 and Rescues…