News

It wasn’t until Gordana Loleska’s son David was 14 years old that doctors in their native North Macedonia diagnosed his kidney, vision, and hearing problems as Alport syndrome. Although she had known for years that something was wrong, the news that David would battle a lifelong rare disease devastated…

The klotho protein may have brain-protecting effects in amyotrophic lateral sclerosis (ALS), according to a study in mice that may become a basis for developing new therapies. The study, “Klotho Is Neuroprotective in the Superoxide Dismutase (SOD1G93A) Mouse Model of ALS,” was published in the Journal…

A new patient-reported way of measuring shortness of breath in amyotrophic lateral sclerosis (ALS), called the Dyspnea-ALS-Scale or DALS-15, can help doctors identify people who would benefit from noninvasive ventilation, even when their lung function and blood gas tests do not indicate it. DALS-15 also can be…

Differences in how efficiently nerve cells degrade and discard proteins may explain why some motor neurons better resist a buildup of toxic proteins than do others in people with amyotrophic lateral sclerosis (ALS), according to a study in mice. The study, “Stem cell-derived cranial and spinal motor…

People who have a high body mass index or gain weight as they age may be at a lower risk of amyotrophic lateral sclerosis (ALS), according to a large population study done in Norway. Those who were obese or got heavier through time had a more than 30 percent…

Nanoparticles may help enhance the therapeutic potential of otherwise difficult-to-deliver agents, such as curcumin, in diseases affecting the central nervous system, including amyotrophic lateral sclerosis (ALS), a review study shows. The review, “Curcumin-loaded nanoparticles: a novel therapeutic strategy in treatment of central nervous system disorders,” was published in the…

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…