News

Touted as the largest amyotrophic lateral sclerosis (ALS) investigative effort to date, the Answer ALS research program has finished an initial technology infrastructure stage and is ready to embark on its goal of finding more effective ALS treatments and, ideally, a cure. A consortium of nearly two dozen medical…

The U.S. Food and Drug Administration (FDA) has granted orphan drug status to Cytokinetics and Astellas Pharma‘s reldesemtiv for the treatment of amyotrophic lateral sclerosis (ALS), the companies announced. Reldesemtiv, a fast skeletal muscle troponin activator designed to improve muscle function with minimal nerve…

Updated results from the CENTAUR Phase 2 trial show that Amylyx‘s investigational oral treatment, AMX0035, significantly slowed progression in treated amyotrophic lateral sclerosis (ALS) patients with rapidly advancing disease compared to those given a placebo. The trial reached its primary goal shortly after its final patient examination in September. Data show…

Mitsubishi Tanabe Pharma America (MTPA) and Soleo Health have entered into an agreement that will facilitate Soleo sharing real-world data about the use of MTPA’s Radicava (edaravone) in amyotrophic lateral sclerosis (ALS) patients. Radicava was approved for the treatment of ALS by the…

The U.S. Food and Drug Administration has agreed to expand Tiglutik, a liquid oral form of riluzole, to include giving it via a feeding tube to treat amyotrophic lateral sclerosis (ALS), ITF Pharma, the company marketing Tiglutik, announced. The approval of administering the therapy via percutaneous endoscopic…

MediciNova‘s experimental therapeutic ibudilast (MN-166) may be most effective for treating amyotrophic lateral sclerosis (ALS) in people with a shorter history of the disease, namely those diagnosed less than 18 months before receiving the treatment, a new analysis shows. The analysis was presented at the…

Researchers with Orion Corporation presented new data on levosimendan‘s mechanism of action, and potential biomarkers of amyotrophic lateral sclerosis (ALS) progression. The data were recently presented in four posters at the 30th International Symposium on ALS/MND, held Dec. 4–6, in Perth, Australia. Levosimendan, a small molecule that acts…

A new ALS Association partnership with the ALS Investment Fund II seeks to raise $100 million in new funding to advance amyotrophic lateral sclerosis (ALS) therapy development. This second venture fund follows one that originated in the Netherlands and raised $25 million in 2.5 years to support…

With so much recent publicity surrounding gene therapy, it’s no surprise that the topic was a major focus of the recent 2019 NORD Rare Diseases & Orphan Products Breakthrough Summit. From diagnosis and clinical trial design to manufacturing, pricing strategies, and ethical concerns, gene therapy — both its high…

Treatment with oral reldesemtiv appears to slow the deterioration of function — including breathing capacity and muscle strength — in amyotrophic lateral sclerosis (ALS) patients, regardless of whether or not they were taking additional medications such as Radicava (edaravone) or Rilutek (riluzole), a new…