News

Inhibition of the SARM1 gene can prevent the degeneration of nerve cells in the central, ocular, and peripheral nervous system in mice, results from preclinical studies show. These findings provide evidence for the use of small-molecule inhibitors of the SARM1 protein being developed by Disarm Therapeutics as potential disease-modifying therapeutics for…

Mutations in a gene linked to familial amyotrophic lateral sclerosis (ALS), called FUS, affect the ability of motor nerve cells to repair damage to their DNA, a new study shows. These findings suggest that therapies that enhance the repair of DNA damage may be of benefit to some ALS patients.

Denali Therapeutics and Sanofi have joined efforts to develop small molecule inhibitors of the RIPK1 enzyme, a new class of potential therapeutic agents for a range of neurological and inflammatory diseases, including amyotrophic lateral sclerosis (ALS). The collaboration agreement is focused on the clinical development of two…

People with multiple rare mutations in genes known to cause amyotrophic lateral sclerosis (ALS) are significantly more likely to develop this disease at younger ages, a study in Japanese patients reports. The study, “Burden of rare variants in causative genes for amyotrophic lateral sclerosis (ALS) accelerates…

While TDP-43 aggregates are associated with amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases, researchers have found that these protein clumps are also involved in the regeneration of  healthy skeletal muscle. This finding — that abnormal TDP-43 aggregates temporarily form to repair damaged muscle — may open new lines of research into…

Extracts of velvet bean and Indian ginseng, two plants commonly used in the Ayurvedic medicine of India, were found to ease sleep disturbances and hyperactivity in a fruit fly model of amyotrophic lateral sclerosis (ALS), while also rescuing locomotor function. The study, “Standardized phytotherapic extracts rescue anomalous locomotion and electrophysiological responses…

Stem cell therapy seems to be safe in patients with amyotrophic lateral sclerosis (ALS) and clinical trials evaluating its effectiveness are on the way, according to a review study. The review, “Stem cell transplantation for amyotrophic lateral sclerosis,” was published in the journal Current Opinion in…

Integrated care is a definite advantage of multidisciplinary clinics according to amyotrophic lateral sclerosis (ALS) patients, but many complain about travel and mobility impediments as major barriers to attend such clinics, a Canadian study reports. Many patients also look at multidisciplinary clinics as potential ALS patient advocates to inform health…

Treatment with a compound called inosine over 12 weeks increased the levels of the antioxidant urate, a neuroprotective agent, in patients with amyotrophic lateral sclerosis (ALS), according to a pilot trial. The investigational therapy also showed a positive safety and tolerability profile, indicating it might have potential as an…

With the U.S. midterm elections now less than two weeks away, patient advocacy groups are solidly focused on a range of hot-button issues, from the Orphan Drug Tax Credit and affordable health insurance to future funding for rare disease research. Yet “whether Democrats take over the House or Senate,…