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A European Medicines Agency (EMA) committee again has delayed issuing an opinion on whether to grant conditional approval to masitinib, a potential add-on oral therapy for amyotrophic lateral sclerosis (ALS). AB Science, the therapy’s developer, now expects a recommendation in the second quarter of this year. The…

An early-stage clinical trial evaluating CK0803, Cellenkos‘ regulatory T-cell-based therapy for people with amyotrophic lateral sclerosis (ALS), has completed dosing its first patient group, the company announced in a press release. The six patients, all adults, were treated at the Columbia University Irving Medical Center, in New…

On the heels of promising HEALEY-ALS Phase 2 trial data, Prilenia Therapeutics plans to launch a pivotal Phase 3 study of pridopidine, its candidate amyotrophic lateral sclerosis (ALS) treatment, in the second half of this year. Relative to a placebo, pridopidine was associated with slower disease progression,…

Dewpoint Therapeutics has received a grant from the Target ALS Foundation to advance the development of small molecules called c-mods, which will target the TDP-43 condensates thought to drive most cases of amyotrophic lateral sclerosis (ALS). Condensates are borderless compartments in cells that can cause cellular processes…

Blocking certain inflammatory signaling pathways may be one way to reverse nerve cell alterations that drive the symptoms of amyotrophic lateral sclerosis (ALS), according to preclinical research using a mouse model of the disease. In the mice, an ALS-associated mutation in the FUS gene caused caused motor neurons — the…

Up to three years of daily treatment with the investigational therapy IPL344 was well tolerated and slowed disease progression among adults with amyotrophic lateral sclerosis (ALS), according to top-line data from a small Phase 1/2a clinical trial. Preliminary efficacy analyses also indicated that IPL344 might be able to…

Note: This story was updated Jan 24, 2024, to correct that Radicava and Radicava Oral Suspension are available in Switzerland but not in other European countries. FAB122, an oral formulation of edaravone developed by Ferrer, failed to slow disease progression or extend survival in adults with amyotrophic lateral…

The proportion of people diagnosed with familial amyotrophic lateral sclerosis (ALS) among all cases is 8%, according to a pooled analysis of studies published worldwide. Still, data show there was a wide variation of estimates across the studies based partly on geographical differences, study design, the definition of familial…

CL2020, a stem cell-based therapy that was being developed by the Life Science Institute, part of Mitsubishi Chemical, was found to be safe and  tolerated well by five people with amyotrophic lateral sclerosis (ALS) who took part in a Phase 2 clinical study. The investigational therapy, which involved…