Studies in animal models investigating mitochondrial abnormalities in amyotrophic lateral sclerosis (ALS) found that therapies targeting mitochondria — the powerhouses of cells — are consistently effective in prolonging survival, a review concluded. The review study, “Targeting…
News
Like many couples, Joe and Linda Lacroix of Milton, Vermont, spent much of their lives working, raising families, maintaining their home, and planning for the future. Unlike most, however, there was always a dark cloud looming on their horizon — a gene for Huntington’s disease carried by Joe’s mother.
Skin cells from people with amyotrophic lateral sclerosis (ALS) have altered metabolisms and increased levels of a type of cellular stress called oxidative stress, a new study shows. This may indicate new treatment targets for ALS. The study, “ALS skin fibroblasts reveal oxidative stress and ERK1/2-mediated…
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…
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