The two most common measures used in amyotrophic lateral sclerosis (ALS) assessments — the ALS functional rating scale-revised (ALSFRS-R) and forced vital capacity (FVC) — can predict survival more accurately when used together, according to a recent study. Alone, these measures had a similar predictive ability, but the weak…
Actemra (tocilizumab), an approved treatment for rheumatoid arthritis, was found to be safe and well-tolerated, and to reduce a key marker of inflammation in amyotrophic lateral sclerosis (ALS) patients with evidence of systemic inflammation in a Phase 2 study. Trial results did not demonstrate that Actemra could slow ALS disability…
Mitsubishi Tanabe Pharma America (MTPA) and Target ALS welcome nominations for the Target ALS Rebecca Luker Courage Award, honoring those who have demonstrated courage and made a positive impact on the amyotrophic lateral sclerosis (ALS) community over the past year. “We have dedicated ourselves to a singular…
Dosing has begun in a clinical trial of WVE-004, Wave Life Sciences‘ investigational therapy for amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) associated with C9orf72 gene mutations. “ALS and FTD are devastating illnesses where therapeutic progress has been extremely limited. Advancing discovery and development of new treatments…
Verge Genomics and Eli Lilly have entered into a three-year collaboration to find and develop therapies for amyotrophic lateral sclerosis (ALS). Verge will focus on discovering and validating potential new therapeutic targets, while Lilly will select up to four candidates to advance through clinical testing with a goal of…
A branch of the European Medicines Agency (EMA) has recommended that Prilenia’s investigational therapy pridopidine be designated an orphan medicine to treat people with amyotrophic lateral sclerosis (ALS). Medicines with the potential to become safe and effective treatments for rare, life-threatening, or chronically debilitating conditions affecting no more than one…
Note: This story was updated June 30, 2021, to clarify that Exservan is placed on top of the tongue, not under it. Specialty pharmacy services for Exservan (riluzole), a twice-daily film that dissolves when placed on the tongue and approved to treat amyotrophic lateral sclerosis…
CRISPR Therapeutics and Capsida Biotherapeutics have partnered to develop gene-editing therapies for familial amyotrophic lateral sclerosis (ALS) and Friedreich’s ataxia. Capsida specializes in designing virus-based means of delivering therapies to specific cells, while CRISPR’s expertise lies in gene-editing technology, particularly the CRISPR gene-editing system. “Bringing together…
AcuraStem has been awarded a $1 million grant from the U.S. Department of Defense (DOD) to advance AS-202, a potentially disease-modifying therapy for sporadic amyotrophic lateral sclerosis (ALS), according to a press release. The candidate therapy is designed to increase the number of lysosomes — cell compartments…
AB Science will suspend recruitment and treatment in all studies involving masitinib, a tyrosine kinase inhibitor being investigated for amyotrophic lateral sclerosis (ALS) and several other conditions. The voluntary suspension follows the identification of a potential risk of ischemic heart disease — a condition of recurring chest…
