News

A machine learning algorithm has shown that depression and a perceived lower quality of life are significant predictors of high caregiver burden among those who care for patients with amyotrophic lateral sclerosis (ALS), a study has found. The study, “Prediction of caregiver burden in amyotrophic lateral sclerosis:…

The number of treatments for children with rare diseases has grown over the past decade, according to a new study. However, despite the increase, nearly 7,000 rare diseases are still lacking treatment. And federal incentives to boost treatment development for these rare diseases have primarily focused not on creating new…

There are differences in the way the fluid around the brain and spinal cord moves in people with amyotrophic lateral sclerosis (ALS), and understanding these could have implications for the development of future therapies, a study suggests. The study, “Non-invasive MRI quantification of cerebrospinal fluid dynamics…

A gene editing technology based on CRISPR was able to slow the progression of amyotrophic lateral sclerosis in a mouse model, a new study showed, demonstrating the approach’s potential for gene therapy in people with ALS. The study, “Treatment of a Mouse Model of ALS by…

The European Medicines Agency (EMA) granted orphan drug status to Cytokinetics‘ reldesemtiv for the treatment of amyotrophic lateral sclerosis (ALS), the company announced. The move follows fast track designation granted in December by the U.S. Food and Drug Administration for the same indication, and provides the…

In recognition of Rare Disease Day Feb. 29, Bionews Services launched a social media campaign last month asking patients to describe what makes them rare. Running Feb. 7–29, the #WhatMakesMeRare campaign was aimed at uplifting people with rare diseases by encouraging them to share their stories and perspectives. The…

A zebrafish model that reproduces key amyotrophic lateral sclerosis (ALS) symptoms and characteristics, including protein clumping, when exposed to blue light could aid in understanding disease mechanisms and in developing new treatments. The model was described in the study, “Optogenetic modulation of TDP-43 oligomerization accelerates ALS-related…

Beginning on Feb. 29, Rare Disease Day, chapters from notable scientific books and clinical review articles covering rare disorders will be available free-of-charge from Elsevier. The offer runs through April 30, and aims to supports work by researchers and clinicians into a better understanding of and treatments for rare diseases, as well…

The Medidata Institute and Project ALS have announced a research partnership aimed at better understanding amyotrophic lateral sclerosis (ALS) and developing new and personalized treatment approaches. With a focus on rare diseases, the institute works with scientists, regulatory agencies, patient advocates, life science companies, and research…

Astrocytes — cells of the nervous system cell that provide support to neurons — were seen to help protect motor neurons from damage caused by toxic protein clumps in a cell model of amyotrophic lateral sclerosis (ALS). The study reporting this finding, “Distinct responses of neurons…