Agamree (vamorolone) - Santhera
Integrating Genetic Modifier Genotype With Serum Proteomics in Duchenne Muscular Dystrophy Clinical Trials Links LTBP4 Genetic Modifier to IL-23/CD93 Pathways in Muscle. (PubMed, Am J Med Genet A) - Aug 20, 2026 - P2, P2a, P2b | "Participants in clinical trials (VBP15-002/003 [n = 48]; VBP15-004 [n = 121]; DNA available for n = 110) were genotyped for eight published genetic modifier loci, and associations of genotypes with baseline motor function defined via an age-adjusted linear model...In contrast, the DYNLT5 genotype was associated with proteosome and chaperonin pathways. Trial Registration: clinicaltrials.gov identifier: NCT02760264, NCT02760277, NCT03439670." 
Journal • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy • CD93 • IL23A • IL6
http://www.ncbi.nlm.nih.gov/pubmed/42613652
 
Daniele Sabbatini; Elena Pegoraro; Eric P Hoffman; Jesse Damsker; John van den Anker; Jyoti K Jaiswal; Kanneboyina Nagaraju; Laura Hagerty; Lauren Morgenroth; Luca Bello; Michael Ziemba; Michela Guglieri; Paula R Clemens; Surajit Bhattacharya; Utkarsh J Dang; Yetrib Hathout; Yuan Fang
 
Aug 20, 2026
 
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