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…
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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…
Factors like head trauma or fracture, electric shock, playing some competitive sports, drinking water from private wells, use of herbicides for gardening, and taking specific dietary supplements all may be associated with the development of amyotrophic lateral sclerosis (ALS), according to a new study. However, larger studies are required…
A neurotoxic amino acid called β-N-methylamino-L-alanine (BMAA) fed to vervet monkeys caused damage to the spinal cord similar to what is seen in amyotrophic lateral sclerosis (ALS), potentially making them a much-needed new animal model for research, a study reported. Adding L-serine, a non-toxic amino acid, to their diet…
Starting a 501(c)(3) tax-exempt nonprofit isn’t easy, but the National Organization for Rare Disorders gave a few tips for those looking to begin the complex process in its Feb. 20 webinar. William Whitman…
Aiforia Technologies and NeuroScience Associates (NSA Labs) are collaborating in an effort to learn more about how amyotrophic lateral sclerosis (ALS) affects brain function. The project leverages NSA Lab’s neuroscience expertise and Aiforia’s artificial intelligence (AI) know-how. It came about after the donation of the entire…
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