Cromolyn sodium — a U.S. Food and Drug Administration (FDA)-approved anti-inflammatory medication — delayed disease onset and prevented motor neuron degeneration and loss in a mouse model of amyotrophic lateral sclerosis (ALS), researchers report. These neuroprotective effects were accompanied by a decrease in inflammation, highlighting the potential…
Mitsubishi Tanabe Pharma America (MTPA) has enrolled a first patient in a Phase 3 clinical trial of an oral suspension formulation of edaravone in treating amyotrophic lateral sclerosis (ALS). An intravenous form of edaravone (given directly into the bloodstream), developed by Mitsubishi Tanabe Pharma and sold under the brand…
Kadimastem’s experimental cell therapy — AstroRx — may slow disease progression in patients with amyotrophic lateral sclerosis (ALS), according to interim results in a first group of patients treated in a Phase 1/2a study. AstroRx consists of functional healthy astrocytes — cells that support and protect neurons…
Sativex, a cannabis-based product, may help ease spasticity (stiff muscles) in people with amyotrophic lateral sclerosis (ALS), a German real-world study suggests. The study, “Real world experience of patients with amyotrophic lateral sclerosis (ALS) in the treatment of spasticity using tetrahydrocannabinol:cannabidiol (THC:CBD),” was published recently in…
An experimental gene therapy based on RNA interference (RNAi) shows potential to treat patients with familial amyotrophic lateral sclerosis (ALS) caused by mutations in the superoxide dismutase 1 (SOD1) gene, according to results of a preclinical study in nonhuman primates. The study, “Safe and effective superoxide…
The U.S. Food and Drug Administration (FDA) has accepted for review the new drug application (NDA) of Biohaven Pharmaceuticals’ BHV-0223, a sublingual (dissolved under the tongue) form of riluzole, as a treatment for amyotrophic lateral sclerosis (ALS). Biohaven has begun enrollment of ALS patients for its Phase 2/3…
Sensory nerves carrying mutations in the TDP-43 or SOD1 genes — associated with the development of a…
The prevalence of motor nerve diseases — including amyotrophic lateral sclerosis (ALS) — is on the rise worldwide, mostly because of an aging population. The disease burden is greatest in high-income countries, according to a global analysis of data from 1990 to 2016. The study, “Global, regional,…
While TDP-43 aggregates are associated with amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases, researchers have found that these protein clumps are also involved in the regeneration of healthy skeletal muscle. This finding — that abnormal TDP-43 aggregates temporarily form to repair damaged muscle — may open new lines of research into…
Stem cell therapy seems to be safe in patients with amyotrophic lateral sclerosis (ALS) and clinical trials evaluating its effectiveness are on the way, according to a review study. The review, “Stem cell transplantation for amyotrophic lateral sclerosis,” was published in the journal Current Opinion in…
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