For a man whose passion involved solving the mysteries of the universe, Stephen Hawking remained a mystery to many. First and foremost he was a British physicist, earning accolades and respect for his many contributions to science. Secondly, as a person living with ALS, his fame came…

After eight years of research, scientists have finally discovered how TDP-43 and hnRNP A1 — two proteins that have been linked to amyotrophic lateral sclerosis (ALS) — work together to promote the disease. TDP-43 was found to bind to the hnRNP A1 RNA sequence and promote production of a…

Difficulty sharing vast amounts of information — the so-called big data — can slow the pace of scientific advancement, experts say. A University of Washington team has come up with a way to help neuroscience researchers share huge swaths of MRI information about ALS and similar disorders. The information deals…

Three organizations that are fighting neurodegenerative disorders have teamed up to challenge researchers around the world to come up with new treatment approaches. The ALS Association, the Huntington’s Disease Society of America (HDSA) and Teva Pharmaceuticals have started the crowdsourcing effort, known as the Teva CNS Target Identification…

This 2014 Roundabout U video is all about Todd Walker from Murray, Kentucky. Todd shares his ALS story, which began when he was suddenly unable to exercise like usual. At first, doctors thought Todd was suffering from depression as he had just lost his father but as his symptoms began…

Scientists have discovered a key role for the ALS-linked protein known as fused in sarcoma (FUS) in a system that is crucial to regulating gene expression. The study, “FUS Regulates Activity of MicroRNA-Mediated Gene Silencing,” was published in the journal Molecular Cell. FUS is a DNA/RNA-binding protein involved in repairing…

Syracuse University researchers have obtained further insight into the protein UBQLN2, whose mutations have been associated with the development of amyotrophic lateral sclerosis (ALS). The study, “Ubiquitin Modulates Liquid-Liquid Phase Separation of UBQLN2 via Disruption of Multivalent Interactions,” was published in the journal Molecular Cell. There are many…

High levels of a protein that transports vitamin A through the body helps protect against the development and progression of ALS, a German study suggests. The protein, retinol binding protein 4 (RBP4), is secreted by fat tissue. The study’s findings suggest it is a potential target for a therapy that could…

In his 10 months on the job, Commissioner Scott Gottlieb of the U.S. Food and Drug Administration is earning praise for his efforts to make clinical trials for new therapies more flexible and responsive to the needs of rare disease patients. From cystic fibrosis to epidermolysis bullosa, the FDA…

Researchers have identified genes that promote amyotrophic lateral sclerosis (ALS), using the gene-editing technology CRISPR-Cas9. The findings represent not only another piece of information to understand the molecular mechanisms triggering ALS, but also support the newly identified genes as potential targets for future therapeutics. The study “CRISPR–Cas9 screens in human cells…