New data show that blood-based measures, including levels of markers of nerve damage and chemical DNA modifications, can help predict which individuals with amyotrophic lateral sclerosis (ALS) will have faster disease progression over time. However, because the disease course may be altered with Radicava or Radicava ORS —…
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Note: This story was updated March 6, 2024, to correct the poster was presented by Brainstorm’s executive vice president and chief development officer Bob Dagher. A pivotal, Phase 3b clinical trial is planned to test the cell-based therapy NurOwn (debamestrocel) in people with amyotrophic lateral sclerosis (ALS),…
While the most recent guidelines for treating amyotrophic lateral sclerosis (ALS) recommend that patients receive three available treatments together, as soon as possible, a new analysis of U.S. data shows that most individuals with the progressive disease don’t get this combination in clinical practice. The analysis found that most…
Treatment with NeuroSense Therapeutics’ PrimeC was found to extend patients’ time without complications or death, and to lead to clinically meaningful effects on quality of life, among those with amyotrophic lateral sclerosis (ALS). The new data comes from the PARADIGM Phase 2b study (NCT05357950), a fully enrolled…
OrphAI Therapeutics’ experimental therapy AIT-101 has been awarded orphan drug status in the European Union for amyotrophic lateral sclerosis (ALS), following a similar designation granted in the U.S. last year. In Europe, orphan drug status is given to medicines designed to treat life-threatening or chronically debilitating conditions affecting…
Treatment with CNM-Au8 was shown to significantly prolong survival — relative to the natural history of the disease — among people with advanced amyotrophic lateral sclerosis (ALS) who took part in two expanded access programs (EAP), also known as compassionate use programs. The findings from these EAPs, which…
An advisory committee of the European Medicines Agency (EMA) has recommended that Biogen’s Qalsody (tofersen) be approved in the European Union under exceptional circumstances to treat adults with amyotrophic lateral sclerosis (ALS) associated with SOD1 gene mutations. The positive opinion from the Committee for Medicinal Products for Human Use,…
Health Canada has issued a notice of noncompliance-withdrawal saying it won’t approve the oral therapy masitinib as an add-on treatment for amyotrophic lateral sclerosis (ALS). Its developer, AB Science, can submit a request for reconsideration within the next month, which it plans to do, it announced in…
ProJenX has been given the green light to expand its ongoing Phase 1 clinical trial of prosetin for amyotrophic lateral sclerosis (ALS) across the Atlantic Ocean to testing sites in Europe. The PRO-101 trial (NCT05279755), launched in 2022, is a three-part study evaluating prosetin’s safety, tolerability, and pharmacological…
Denali Therapeutics’ SAR443820 (DNL788) treatment candidate — now in late-stage clinical testing for amyotrophic lateral sclerosis (ALS), per the company’s website — has failed to significantly slow disease progression in adults in a Phase 2 trial. The experimental therapy “did not meet the primary endpoint of change” in outcomes…
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