Treatment with Relyvrio (sodium phenylbutyrate and taurursodiol) in the CENTAUR trial significantly extended the median survival — by 10.4 months — of amyotrophic lateral sclerosis (ALS) patients compared with an historical patient control group, a post-hoc analysis of study data reported. This finding more than doubles the 4.8…
The National Institutes of Health (NIH) has awarded about $45.1 million toward an expanded access program (EAP) of CNM-Au8, an oral therapy candidate for amyotrophic lateral sclerosis (ALS). The EAP, commonly referred to as compassionate use, will make CNM-Au8 available to U.S. patients who aren’t eligible for clinical…
An advisory committee of the European Medicines Agency has maintained its recommendation not to approve AMX0035 for amyotrophic lateral sclerosis (ALS). In its initial assessment issued in June, the Committee for Medicinal Products for Human Use (CHMP) said data from the CENTAUR Phase 2 trial (NCT03127514), which formed…
The Barrow Neurological Institute has received $16.7 million from the National Institutes of Health (NIH) to help coordinate a nationwide repository of clinical data of people with amyotrophic lateral sclerosis (ALS). The Access for All in ALS (ALL ALS) Consortium will serve as a repository of biological samples and clinical…
PrimeC, NeuroSense Therapeutics‘ investigational, fixed-dose combination therapy for amyotrophic lateral sclerosis (ALS), significantly boosted the survival of patient-derived motor neurons to levels similar to healthy controls in a lab study. The independent study was led by Justin Ichida, PhD, a professor of stem cell biology and regenerative…
Amyotrophic lateral sclerosis (ALS) patients not eligible to participate in clinical trials of pridopidine — a small molecule developed by Prilenia Therapeutics to slow disease progression in ALS — will soon be able to receive the experimental therapy via an expanded access program (EAP). EAPs, also known…
The European Medicines Agency (EMA) is now expected to issue a decision in the first months of 2024 on the conditional approval of masitinib as an add-on oral therapy for amyotrophic lateral sclerosis (ALS). The deadline extension follows a request, seeking more time, from AB Science, the therapy’s developer,…
People with amyotrophic lateral sclerosis (ALS) are not at a higher risk of complications due to a spinal tap than are healthy individuals or those with other neuromuscular disorders, according to a small U.S. study. Findings show that ALS patients have a relatively lower risk for such adverse events,…
Early treatment with Radicava (edaravone), an approved therapy for amyotrophic lateral sclerosis (ALS), significantly reduced the risk of hospitalization, need for ventilatory support, or death compared with delaying treatment for six months. That’s according to analyses of data from Study 19 (NCT01492686), the Phase 3 clinical trial…
A grant given to a researcher at the University of Albany in New York will support further work into specific RNA molecules, called RNA aptamers, that have shown early promise in treating amyotrophic lateral sclerosis (ALS). The $768,825 U.S. Department of Defense award to Li Niu, PhD, a chemistry…
