Work into developing molecules that block microRNA-155 (miR-155) as potential treatments for amyotrophic lateral sclerosis (ALS) has advanced to the stage of testing candidate molecules in animal models of the disease. Initiated last year, the miR-155 project is a collaboration between scientists at Regulus Therapeutics and Brigham…
ALS research into miR-155 blockers advances to animal testing stage
A Phase 3 clinical trial that’s testing FNP122, Ferrer’s oral formulation of edaravone, in people with amyotrophic lateral sclerosis (ALS) is now rolling participants into its open-label extension study. The ADORE trial (NCT05178810) is testing FNP122 against a placebo in about 300 patients across Europe whose…
VRG50635, an experimental oral therapy being developed by Verge Genomics for amyotrophic lateral sclerosis (ALS), was found to be safe and well-tolerated in healthy volunteers, new Phase 1 trial data show. Top-line data from the trial (ISRCTN14792372) also showed a promising tolerability and pharmacological profile supporting once-daily…
The Committee for Medicinal Products for Human Use (CHMP) has issued an opinion opposing Amylyx Pharmaceuticals’ bid for conditional European Union approval of AMX0035, its oral treatment to slow the progression of amyotrophic lateral sclerosis (ALS). The negative recommendation from the advisory committee, an arm of the…
As I consider the progress we’ve made on ALS, I can’t help wondering if our overall awareness will continue to grow and lead us to even more sensitivity and advocacy. What real action will take place? Will initiatives be developed and sustained? Will we patients be closer to…
The first healthy volunteer has been dosed in a Phase 1 clinical trial evaluating VTX3232, Ventyx Biosciences’ investigational oral therapy for amyotrophic lateral sclerosis (ALS) and other neurodegenerative conditions. Top-line data from the trial are expected in the first half of 2024. “VTX3232 is our fourth internally discovered…
Magnetic stimulation restored the health of lab-grown motor neurons — the muscle-controlling nerve cells that die in amyotrophic lateral sclerosis (ALS) — derived from people with familial ALS, according to a proof-of-concept study. The approach improved several biological processes in motor neurons, including the movement of cellular components along…
Ever since my husband Todd’s ALS led to his paralysis, it has been hard for him to get comfortable. This is especially true now that he wears a mask nearly 24/7 for noninvasive ventilation support. For years, I’ve jumped into action when he calls me to swat a…
Albrioza, the approved treatment for amyotrophic lateral sclerosis (ALS) formerly known as AMX0035, is one giant step closer to being eligible for reimbursement under public drug plans in Canada, according to its developer, Amylyx Pharmaceuticals. Amylyx has reached an agreement with the pan-Canadian Pharmaceutical Alliance, or pcPA —…
PEG10, an ancient, virus-like protein that supports the development of the placenta, appears to also participate in the nerve cell loss associated with amyotrophic lateral sclerosis (ALS), researchers discovered. ALS-causing mutations in the UBQLN2 gene were found to prevent the proper degradation of PEG10. This led to a…
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