Mutations that affect a particular molecular pathway essential to protein production further support reports of a link between amyotrophic lateral sclerosis (ALS) and spinal muscular atrophy (SMA). The study detailing this finding, “The neurodegenerative diseases ALS and SMA are linked at the molecular level via the ASC-1 complex,”…
A gene therapy effectively and safely lowered the production of a key amyotrophic lateral sclerosis (ALS) protein called superoxide dismutase 1 (SOD1) in primates, according to a new study. “This level of silencing, coupled with the lack of adverse effects, suggests that this approach to treating ALS should be…
Specific proteins analyzed in cerebral spinal fluid and blood samples may help to diagnose amyotrophic lateral sclerosis (ALS) and predict patient outcomes, researchers report. Cerebral spinal fluid (CFS) protein patterns helped to identify 94% of ALS patients in their study, and the levels of three specific proteins were seen to…
The prevalence of motor nerve diseases — including amyotrophic lateral sclerosis (ALS) — is on the rise worldwide, mostly because of an aging population. The disease burden is greatest in high-income countries, according to a global analysis of data from 1990 to 2016. The study, “Global, regional,…
To bring attention to the plight of teens touched by amyotrophic lateral sclerosis (ALS), Mitsubishi Tanabe Pharma America (MTPA) is calling for artistic submissions from young people who have a family member with the disease. Called ALSO US, the new initiative is open to U.S. teenagers and young…
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…
