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…
A Phase 3 clinical trial evaluating Orphazyme’s investigational oral therapy arimoclomol for amyotrophic lateral sclerosis (ALS) has failed to meet its primary and key secondary goals. While no safety issues were reported in the trial (NCT03491462), arimoclomol did not significantly extend patients’ lives nor…
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 Phase 2 clinical trial evaluating oral RT001 — Retrotope’s experimental synthetic fatty acid — as a treatment of amyotrophic lateral sclerosis (ALS) is fully enrolled, the company announced. The enrollment target of 40 patients was met, and exceeded, ahead of its scheduled six weeks. Dosing began…
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…
A committee with the European Medicines Agency favors designating Seelos Therapeutics‘ investigational therapy SLS-005 (trehalose) an orphan drug as a potential treatment of amyotrophic lateral sclerosis (ALS), the company announced in a press release. The opinion, issued by the Committee for Orphan Medicinal Products (COMP), will now go to the…
National Amyotrophic Lateral Sclerosis Awareness Month, recognized in the U.S. each May since 1992, continues to unite patients, family, friends, and advocacy groups around shared goals of raising public awareness and research support. This year’s efforts continue to recognize and support those living with…
Registration is now open for fundraising events at four championship golf courses, whose fees will be used to fund research, care, and advocacy for neuromuscular disorders such as amyotrophic lateral sclerosis (ALS) and muscular dystrophy. According to 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…
Applications for a $60,000 one-year grant are now open for researchers working to discover or validate biomarkers for amyotrophic lateral sclerosis (ALS) or a similar disorder. Those interested in trying for this award need to submit a letter of intent as to their project’s nature and intent by May 7.
