Copper-ATSM Prevented Motor Neuron Loss in SALS Mouse Model

Copper-ATSM — a compound originally used as an imaging agent — prevented motor neuron loss and reduced brain inflammation in a mouse model of sporadic amyotrophic lateral sclerosis (ALS), a study showed. The study, titled “Neuroprotective effect of CuATSM on neurotoxin-induced motor neuron loss in…

A Phase 2b clinical trial assessing the efficacy and safety of H.P. Acthar Gel, an experimental injectable therapy for amyotrophic lateral sclerosis (ALS), has enrolled half of its anticipated participants so far and is still open for recruitment. The trial, called PENNANT (NCT03068754), intends to enroll roughly…

A violinist with vasculitis, two Texas politicians and a pharmaceutical company whose marijuana-derived therapy helps kids with Dravet syndrome were among winners of the 2019 Rare Impact Awards. Officials of the National Organization for Rare Disorders (NORD) presented the awards during a June 22 dinner attended by…

Scientist Jingsong Zhou, PhD, has received a $2.88-million grant from the National Institutes of Health (NIH) to study how amyotrophic lateral sclerosis (ALS) affects multiple organs beyond the central nervous system (CNS). “We are ultimately working to find some potential therapeutic means to treat the disease with a…

Multidisciplinary care with timely use of respiratory and feeding support contributes to increased survival for people with amyotrophic lateral sclerosis (ALS), a single-center study in Spain finds. The study, “Survival benefit of multidisciplinary care in amyotrophic lateral sclerosis in Spain:…

Europe’s umbrella organization for 800 rare disease associations has developed a sweeping initiative to help the continent’s 30 million rare disease patients and their caregivers learn about their conditions, find assistance and receive treatment. Eurordis-Rare Diseases Europe hopes to improve the current piecemeal treatment and support program with a holistic,…

Vitamin D supplementation can help overcome low levels of the vitamin often detected in people with amyotrophic lateral sclerosis (ALS). Still, such an alternative therapeutic approach has no meaningful clinical effect on progression of ALS-associated motor symptoms, a study suggests. The study, “…

High glucose levels might compensate for the toxic effects of an accumulation of TDP-43 — a widely expressed nuclear protein that binds both DNA and RNA — and protect neurons in amyotrophic lateral sclerosis (ALS), a study in a fly model of the disease suggests. The study,…

Where were you on Feb. 28? Did you know that it was Rare Disease Day? Don’t feel bad if you missed it because I did too. But I shouldn’t have. Because Rare Disease Day, along with the international events around it, represented a major change in perspective toward finding…

Caregivers of people with amyotrophic lateral sclerosis (ALS) and progressive muscular atrophy (PMA) say a Dutch support program — one based on Acceptance and Commitment Therapy — empowered them to make better choices in line with their own needs, and to cope with distress, a study shows. The…