People living with amyotrophic lateral sclerosis (ALS) recently shared their views on the importance of new therapies and incremental gains, and the risks they are willing to take with U.S. Food and Drug Administration (FDA) and industry officials in a virtual meeting hosted by The ALS Association. The…
Researchers at the University of Texas at Dallas (UT Dallas) have received an award from the U.S. Army Medical Research Acquisition Activity to advance their preclinical research of an innovative CRISPR-based gene editing approach to treat one of the most common causes of familial amyotrophic lateral sclerosis (ALS). The two-year…
Cytokinetics has reaffirmed its partnership with The ALS Association in the fight against amyotrophic lateral sclerosis (ALS) by continuing to support events and initiatives throughout 2021. The muscle biology biopharmaceutical company also is working to advance the clinical development of its experimental therapy reldesemtiv, designed to improve…
Researchers at the University of Queensland in Australia have begun preclinical studies of macimorelin, Aeterna Zentaris’ approved oral diagnostic, as a potential treatment for amyotrophic lateral sclerosis (ALS). The preclinical development program, led by Frederik Steyn, PhD, is part of a recent agreement between university researchers and…
Note: This story was updated Sept. 21, 2021, to note that 18% of patients, not 80%, experienced increases in the MUNIX(4) sum score at some point during the study. Participants in a placebo-controlled Phase 2 clinical trial of CNM-Au8, Clene Nanomedicine’s investigational oral therapy for amyotrophic…
SOL-257, an experimental gene therapy developed by SOLA Biosciences, delayed disease progression and prolonged the lifespan of a mouse model of amyotrophic lateral sclerosis (ALS). The gene therapy is designed to reduce the abnormal TDP-43 protein that forms toxic clumps in the cells of ALS patients. “Targeting only…
Jan Veldink, a neurologist from the Netherlands whose work in the genetics of amyotrophic lateral sclerosis (ALS) has led to important discoveries about disease risk, won the 2021 Sheila Essey Award, according to a blog post from the ALS Association. The $50,000 award was presented at the recent…
A combination of guanabenz — an approved therapy for high blood pressure — plus riluzole slowed disease progression in adults with early amyotrophic lateral sclerosis (ALS), particularly those with bulbar onset disease, a form of ALS, according to data from a Phase 2 clinical trial. Despite these promising benefits, the…
People with very rapidly progressing familial amyotrophic lateral sclerosis (ALS) caused by mutations in the SOD1 gene may gain early access to Biogen’s experimental therapy tofersen by mid-July, the company announced. This marks the first part of Biogen’s planned access program for tofersen, and will begin after patients initially assigned…
Editor’s note: The ALS News Today team is providing in-depth coverage of the 2021 Virtual AAN Annual Meeting, April 17–22. Go here to read the latest stories from the conference. Amyotrophic lateral sclerosis (ALS) patients require walking aids, ventilation support, and/or speech generation devices about 1.5 years after starting treatment with…
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