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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…

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…

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. In a Phase 1 trial for amyotrophic lateral sclerosis (ALS), the investigational therapy AP-101 — designed to ease…

The HEALEY ALS platform trial, which is evaluating the safety and efficacy of several potential treatments for amyotrophic lateral sclerosis (ALS), has passed the 50% participant enrollment target for its first three regimens. The investigational treatments are UCB’s zilucoplan, Biohaven Pharmaceuticals’ verdiperstat, and Clene Nanomedicine’s CNM-Au8. A fourth candidate…

“Have you ever participated in clinical trials?” That’s a common question among ALS patients. What’s my standard answer? “It’s complicated. I’m participating in several studies, but even though I’d like to, I haven’t been in any clinical trials. I’m excluded because I’ve had ALS for too many years.” ‘What? Tell…

Mitsubishi Tanabe Pharma America (MTPA) is taking proactive measures to address patient safety in its REFINE-ALS biomarker study during the COVID-19 pandemic. The company announced revisions to the study’s protocol that give participants the option to participate virtually, through the use of telemedicine (medical care delivered virtually) and…

Cytokinetics is preparing a global Phase 3 trial that will enroll more than 500 people with early amyotrophic lateral sclerosis (ALS) to test its investigational therapy reldesemtiv in slowing progressive muscle weakness, the company announced. Details from the trial design were shared at the 31st International Symposium…

IPL344 is an experimental treatment for amyotrophic lateral sclerosis (ALS) that is being developed by Immunity Pharma to slow disease progression. How does IPL344 work? ALS is a progressive disease of the nervous system characterized by the death of motor neurons — nerve cells that control voluntary muscles. The…