Convelo Therapeutics Inc. has presented data on their 3-β-hydroxysteroid-Δ8,Δ7-isomerase (EBP) inhibitor CVL-1001 as a remyelinating compound for treating multiple sclerosis.
Sovargen Co. Ltd. inked a $550 million license deal with Angelini Pharma SpA, granting Angelini development and commercialization rights to SVG-105, a novel antisense oligonucleotide drug candidate in preclinical development as a potential treatment for intractable epilepsy.
Chineses researchers investigated the relationship between formyl peptide receptor 1 (FPR1) expression and neurodegeneration in multiple sclerosis (MS), specifically evaluating the therapeutic potential of the FPR1 antagonist T-0080.
Tevard Biosciences Inc. has presented new preclinical data on the use of therapeutic suppressor tRNAs (suptRNAs) for the treatment of Duchenne muscular dystrophy (DMD) and dilated cardiomyopathy (DCM). The data show potent restoration of full-length functional proteins in models of DMD and DCM caused by titin truncations (DCM-TTNtv).
Up to 71% of people carry at least one pathogenic variant that could contribute to development of a heritable disorder in offspring, but until now, prospective parents often had to undergo multiple tests to understand their risks. Pacific Biosciences of California Inc. (Pacbio)’s expanded Puretarget portfolio provides a quicker and more streamlined solution as it covers all challenging tier 3 genes identified in the American College of Medical Genetics technical standard.
Anaphylaxis rates caused Larimar Therapeutics Inc.’s stock (NASDAQ:LRMR) to take a hit on the latest data from an open-label study with nomlabofusp in the neuromuscular disease Friedreich’s ataxia (FA), but the company is targeting a BLA submission to seek accelerated approval in the second quarter of next year.
Is there a link between cellular senescence and multiple sclerosis (MS) progression? Several presentations at this year’s European Committee for Treatment and Research in Multiple Sclerosis 2025 (ECTRIMS 2025) conference, in Barcelona, which ended Sept. 26, addressed this question
Hager Biosciences LLC has identified orexin receptor and/or κ-opioid receptor dual antagonists reported to be useful for the treatment of cancer, psychiatric, neurological and cardiovascular disorders.
Researchers from Novartis AG reported preclinical efficacy data on VHB-937, an agonist human monoclonal antibody targeting TREM2 in models of neuroinflammation.