With an estimated 1.37 billion inhabitants, India will likely surpass China in five years as the world’s most populous country. That also means it will have more rare-disease patients than any nation. It already has more than twice as many as the 28-member European Union. Harsha K. Rajasimha, a genomics…
News
Continuing its support for a potential therapy for amyotrophic lateral sclerosis (ALS), Australia-based organization FightMND has granted about $700,000 to biopharmaceutical company Collaborative Medicinal Development. The money will fund a randomized, double-blind, placebo-controlled study of CuATSM, one of CMD’s lead investigational therapies. According to a news release, the…
Newly diagnosed amyotrophic lateral sclerosis (ALS) patients often experience poor sleep quality due to symptoms of depression and difficulty turning in bed, a patient survey shows. The study, “Poor Sleep Quality in Patients With Amyotrophic Lateral Sclerosis at the Time of Diagnosis,” was published in the Journal…
Project ALS and Columbia University are launching a new platform with a goal of discovering more useful treatments for amyotrophic lateral sclerosis (ALS) patients, and pushing the most promising candidates to clinical trials. Core, as it…
Aquestive’s new drug application for Exservan — an investigational oral film of riluzole to be used as an add-on therapy for amyotrophic lateral sclerosis (ALS) — has been accepted by the U.S. Food…
The U.S. Food and Drug Administration approved MediciNova’s plans for a pivotal Phase 2b/3 clinical trial that will explore the potential of ibudilast (MN-166) in patients with amyotrophic lateral sclerosis (ALS). Pending the success of the trial, the company hopes the collected clinical data will support the…
Damaged activity of RNA-binding proteins (RBPs) associated with amyotrophic lateral sclerosis (ALS) can be alleviated by inducing autophagy, a process where cells degrade or recycle components that are damaged or no longer needed, a study says. The study, “FUS pathology in ALS is linked to alterations in multiple ALS-associated proteins…
How Common ALS Mutation Damages Cells and Leads to Toxic Protein Being Produced Detailed in Study
The most common genetic cause of amyotrophic lateral sclerosis (ALS) disrupts the shape and workings of a compartment inside a cell’s nucleus, researchers report, detailing a discovery that helps to explain how this mutation prompts cell death in ALS. Abnormalities resulting from this mutation, specifically the length of the…
As many as a third of people with amyotrophic lateral sclerosis (ALS) also have symptoms of Parkinson’s disease, which may be due to differences in brain activity between those with and without these symptoms, a study reports. The study, “Parkinsonian traits in amyotrophic lateral sclerosis…
A possible new target for treating amyotrophic lateral sclerosis (ALS) may be the nonsense-mediated mRNA decay (NMD) pathway, which helps cells remove potentially harmful RNA, like RNA that’s been improperly processed or comes from viruses. Researchers in this early study also suggest that an asthma treatment known as tranilast (marketed in Japan…
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