Nonprofit Works to Raise Rare Disease Awareness in India, Connect with U.S. Groups

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…

A zero-sum game is a situation in which the accumulation of gain is accompanied by a sum of loss of equal value. The net result is always zero. From the get-go, ALS seeks to make that outcome an impossibility. It strives to force a plunging and irreversible deficit. In the…

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…

“If anything can go wrong, it will.” — Murphy’s Law That adage could be the ALS anthem. From the onset of mysterious symptoms to the serpentine-like diagnostic process and worst-case outcome, and by watching your body unfailingly obey his maxim, the prophet Murphy is seemingly ever-present. The…

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…

Recently, I attended an “Ask the Experts” educational seminar sponsored by my local chapter of the ALS Association. It’s an annual event that presents current information on ALS-related topics and allows attendees to exchange ideas with fellow ALS patients. This year’s theme was “ALS clinical trials…

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…