The accumulation of TDP-43, a widely expressed nuclear protein that binds both DNA and RNA, causes RNA instability in nerve cells derived from patients with amyotrophic lateral sclerosis (ALS). That leads to disruption of energy and protein production, and eventually causes cells’ death. Those findings were described in the…
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Researchers have detailed the structural changes that occur in the protein hnRNPA2 when it is mutated in diseases related to amyotrophic lateral sclerosis (ALS). The study, “Mechanistic View of hnRNPA2 Low-Complexity Domain Structure, Interactions, and Phase Separation Altered by Mutation and…
A new guide outlines the benefits of a multidisciplinary team approach to provide the integrated medical and mental treatment that amyotrophic lateral sclerosis (ALS) patients often need. The guide, titled “A Guide to Understanding the Benefits of a Multidisciplinary Team Approach to Amyotrophic Lateral Sclerosis (ALS)…
The active molecule of Petinutin (methsuximide), an anti-epileptic medication, eased motor deficits, extended the lifespan, and showed potent neuroprotective effects in a worm model of amyotrophic lateral sclerosis (ALS), suggesting a potential new treatment strategy for multiple neurodegenerative diseases, a study reports. The study, “α-Methyl-α-phenylsuccinimide ameliorates neurodegeneration…
IONIS-SOD1Rx (BIIB067), an investigational therapy for amyotrophic lateral sclerosis (ALS) developed by Biogen and Ionis Pharmaceuticals, was shown to prolong survival and reverse neuromuscular damage in mouse and rat models…
CannTrust is getting ready to launch a clinical trial to assess the effectiveness of its cannabidiol (CBD) oil capsules on slowing the progression of amyotrophic lateral sclerosis (ALS) and other motor neuron diseases (MNDs). This new study is the result of a collaboration between Canada-based CannTrust and…
Restoring or artificially raising levels of a protein found in the spinal cord, mitofusin 2, worked to prevent nerve degeneration, muscle atrophy, and paralysis in a mouse model of amyotrophic lateral sclerosis, researchers report. As this protein is often depleted in ALS patients — and drops with aging —…
Treatment of amyotrophic lateral sclerosis (ALS) with investigational compound ibudilast (MN-166) as an add-on to Rilutek (riluzole) improves patients’ functional activity, quality of life, and muscle strength, data from MediciNova’s Phase 2 trial show. MediciNova is now recruiting participants for a Phase 1/2 biomarker trial (…
A new Phase 3 trial evaluating levosimendan (also known as ODM-109), an oral treatment by Orion for breathing problems in amyotrophic lateral sclerosis (ALS), has recruited its first patients, the Finnish pharmaceutical company announced. The trial, called REFALS (NCT03505021), aims to investigate the potential benefits of prolonged treatment with levosimendan…
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2 miRNAs Controlling Genes Involved in ALS, and Possible Way of Regulating Them, Identified in Study
Sporadic and familial forms of amyotrophic lateral sclerosis (ALS) are associated with two microRNA molecules that are downregulated — meaning these molecules, involved in the protein production of certain genes, cause less protein to be created —, researchers have discovered. And they found a possible way of “upregulating” these molecules, making…
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