The European Commission has approved Qalsody (tofersen) as a treatment for amyotrophic lateral sclerosis (ALS) associated with mutations in the SOD1 gene, known as SOD1-ALS. Qalsody was specifically granted marketing authorization under exceptional circumstances — a pathway recommended when the benefit-to-risk assessment for a therapy is favorable, but…
News
The Foundation for the National Institutes of Health (FNIH) is launching a public-private partnership to generate the largest openly available data platform in amyotrophic lateral sclerosis (ALS) research, with the goal of accelerating the diagnosis and treatment of the progressive neurodegenerative disease. This five-year initiative will be conducted through…
The Les Turner ALS Foundation, in an effort to boost and diversify clinical trial participation, has written a step-by-step guide to inform people on why clinical research in amyotrophic lateral sclerosis (ALS) needs a diverse pool of participants, how to join clinical research, and what to expect when…
The fourth annual Major League Baseball (MLB) Lou Gehrig Day takes place on June 2, a date set aside to pay tribute to the legendary New York Yankees first baseman whose career was halted by amyotrophic lateral sclerosis (ALS). The Muscular Dystrophy Association (MDA) will again play…
A new documentary about I AM ALS and the “revolutionary movement” begun by the organization’s founders — which will stream globally on May 28 — is seeking to advance awareness of amyotrophic lateral sclerosis (ALS) and other neurodegenerative disorders while inspiring others to fight for change. Called “For…
People with amyotrophic lateral sclerosis (ALS) who continued treatment with NurOwn (debamestrocel) after completing a Phase 3 trial experienced additional benefits with extended treatment, data show. The findings come from 10 adults who received up to six additional doses of NurOwn over nearly one year in an expanded…
By taking advantage of a molecular interaction between the disease-related protein TDP-43 and another, innocuous protein, scientists have found a novel strategy that potentially may be used as a treatment for amyotrophic lateral sclerosis (ALS). “Importantly, this interaction could be key to unlocking a treatment not just for ALS…
NeuroSense Therapeutics is collaborating with PhaseV for insights into how to better design the protocol for the planned Phase 3 trial that will test PrimeC for amyotrophic lateral sclerosis (ALS). A specialist in machine learning technology for clinical trials, PhaseV used data from the ongoing Phase 2b…
Biogen and Ionis Pharmaceuticals have decided to terminate the development of BIIB105, their experimental treatment for amyotrophic lateral sclerosis (ALS), based on data from a Phase 1/2 clinical trial. The therapy resulted in no significant clinical benefit compared with a placebo after six months, and data from…
A new amyotrophic lateral sclerosis (ALS) assessment called delta-FS — defined as the rate of decline over time in the ALS Functional Rating Scale-Revised (ALSFRS-R) — has been proposed as an attractive, easily obtainable tool to monitor disease severity and predict patient prognosis. The assessment also could be used…
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