Researchers at Germany’s University of Würzburg have discovered how a malfunctioning autophagy – a cell’s natural self-degradation and cleanup system – plays a central role in the development of amyotrophic lateral sclerosis (ALS) and other motor neuron diseases. The study, “Plekhg5-regulated autophagy of synaptic vesicles reveals a pathogenic mechanism…
Defects in Cell Cleanup System May Lead to ALS and Other Diseases, Mouse Study Shows
Biohaven Pharmaceutical has dosed the first amyotrophic lateral sclerosis (ALS) patient with its sublingual formulation of riluzole, BHV-0223, in a pivotal Phase 2/3 trial. The trial aims to demonstrate that BHV-0223’s effects are at least equivalent to Rilutek, an approved tablet formulation of riluzole. The trial initiation was supported by…
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What Does ALS Look Like to You?
The other day, while skimming through a list of reader comments on ALS News Today, I ran across one remark that stopped me in my tracks. It was a complaint regarding the photo that accompanies this column. The reader thought the woman in the wheelchair looked too healthy…
Option Care Enterprises, a Chicago-based independent provider of home and alternate treatment site infusion services, has partnered with the ALS Association to offer Radicava (edaravone) treatment from the comfort of their homes. To date, more than 100 amyotrophic lateral sclerosis (ALS) patients are already receiving Radicava — the first…
Following her husband’s frustration with not being able to do up the buttons on his formal work shirts, Maura Horton came up with a simple and practical solution: magnetic buttons. MORE: Seven facts about amyotrophic lateral sclerosis you might find interesting According to a report on Fox News, Horton’s husband Don…
The U.S. Food and Drug Administration (FDA) has given the green light to Biohaven Pharmaceuticals to advance sublingual BHV-0223, which regulates glutamate communication between neurons, into clinical trials for amyotrophic lateral sclerosis (ALS) patients. The FDA’s permission to go ahead with the study followed Biohaven’s submission of an investigational new drug…
Repeated severe trauma to the body or mild injuries to the head — especially at midlife — raise a person’s risk of ALS, a European case-control study from the EURALS consortium shows. The new research, “Trauma and amyotrophic lateral sclerosis: a european population-based case-control study from…
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An ALS Documentary: ‘Mr. Connolly Has ALS’
https://vimeo.com/216244227 This film by Dan Habib is all about the extraordinary life of high school principal Gene Connolly. Connolly was the much-loved principal of Concord High School for 14 years before he was struck by amyotrophic lateral sclerosis (ALS) in 2014. MORE: Explaining the progression…
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…
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