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Children’s National Health System no longer treats just kids. Its Rare Disease Institute, launched in April 2017, has partnered with the National Organization for Rare Disorders (NORD) to become the first of many U.S. “centers of excellence” to look after patients with rare diseases, regardless of age. The effort…

In some neurodegenerative diseases, such as amyotrophic lateral sclerosis (ALS), nerve cells fold their proteins incorrectly, leading to cell death. Using plants, researchers discovered that when defective proteins accumulate in chloroplasts – a plant’s cellular compartment where photosynthesis occurs – it generates a distress signal that produces reparative proteins to…

A nervous system mechanism that helps control heart rate and blood pressure goes into overdrive in patients with a type of ALS that eliminates mobility and sensation in some limbs, a study reports. The control mechanism is known as sympathetic vascular response — with vascular response referring to heart blood vessels.

BrainStorm Cell Therapeutics is expanding the patent portfolio that protects its NurOwn technology for the treatment of several diseases affecting the central nervous system. The latest U.S. patent claim includes Parkinson’s disease and ALS (amyotrophic lateral sclerosis). The announcement by BrainStorm follows a Notice of Allowance issued by…

​Researchers generated motor neurons from  amyotrophic lateral sclerosis (ALS) patients with a mutated gene found in familial forms of the disease. The study sheds new light on the disease mechanism. Transport of materials up and down the motors’ neuron length (axon) keeps nerve cell messages…

Relatives of patients with amyotrophic lateral sclerosis (ALS) have a higher likelihood of developing neuropsychiatric symptoms including schizophrenia, obsessive-compulsive disorder, autism, and alcoholism, research from Ireland shows. The study, “Clustering of Neuropsychiatric Disease in First-Degree and Second-Degree Relatives of Patients With Amyotrophic Lateral Sclerosis,” was published…

Masitinib may potentially help amyotrophic lateral sclerosis (ALS) patients by targeting mast cells, a Uruguayan study shows. The study, “Evidence for mast cells contributing to neuromuscular pathology in an inherited model of ALS,” appeared in JCI insight. ALS, a neurodegenerative disorder, is characterized by…

Researchers have created an atlas that links a set of genes in the DNA to a particular trait or disease, a tool that may advance understanding of certain cell characteristics in neurological diseases like Alzheimer’s, Parkinson’s and amyotrophic lateral sclerosis (ALS). This atlas can be further developed and updated by…

Military veterans in the United States who were deployed to World War II or who handled burning agents during their service show shorter amyotrophic lateral sclerosis (ALS) survival, a new study finds. The research, “Military service, deployments, and exposures in relation to amyotrophic lateral sclerosis survival,” appeared in…

More therapies are now available for the 30 million or so people with rare diseases in the U.S. than ever before, and millions of dollars are being invested in clinical studies that will test new ways of evaluating — and advancing — potential treatments, including the use of natural history…