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…
When I learned I had ALS, my whole world changed, because on that day, I officially became an ALS patient. My husband’s world changed as well because, just as suddenly, he became my caregiver. I appreciate his taking on this challenging responsibility more than he’ll ever know.
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…
Living in the present has always been hard for me. I had such grand dreams for my future. It is hard to come to terms with the fact that I won’t go on a camel trek in the Sahara and spend the night under the most beautiful…
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.
Living with ALS presents many challenges, and staying mentally motivated is right at the top of the list. Being proactive to prevent our emotions from spiraling downward is easier than picking ourselves up after we’ve hit rock bottom. So, I want to share the five things I…
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…
