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,…
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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…
To reflect a new era of commitment and determination in the fight against amyotrophic lateral sclerosis (ALS), The ALS Association Greater New York Chapter has become ALS United Greater New York. The rebranding of the New York City-based nonprofit represents a break…
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…
An initiative at a University of Illinois campus that aims to improve voice recognition software for people with speech difficulties is recruiting adults in the U.S. and Puerto Rico living with amyotrophic lateral sclerosis (ALS). Known as the Speech Accessibility Project, it is led by the Urbana-Champaign…
Verge Genomics has launched a proof-of-concept trial to test whether VRG50635, its oral PIKfyve inhibitor therapy, can be used as a potential treatment for people with amyotrophic lateral sclerosis (ALS). The Phase 1b study will assess the safety and tolerability of escalating doses of VRG50635 — which works…
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