ArticleThe Journal of pharmacology and experimental therapeutics2019
Clinically Advanced p38 Inhibitors Suppress DUX4 Expression in Cellular and Animal Models of Facioscapulohumeral Muscular Dystrophy.
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Article in The Journal of pharmacology and experimental therapeutics, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 54 papers.
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54citing papers in PubMed
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Who cites it
54 citing papers in PubMed, 88 citations in OpenAlex.
- Nanopore Ultra-Long Sequencing for FSHD: From Molecular Diagnosis to Preimplantation Genetic Testing.Genes · 2026Review
- Identification of compounds that repress DUX4 expression in facioscapulohumeral muscular dystrophy.Scientific reports · 2026Article
- A study evaluating differences in 3D upper limb kinematics and surface electromyography measures in adults with and without facioscapulohumeral dystrophy.JSES reviews, reports, and techniques · 2026Article
- Chronic activation of p38α in skeletal muscle causes necrotic changes, but also abolishes expression of MK2, MK3, and MKK6 and the muscle recovers.The Journal of biological chemistry · 2026Article
- Identification of compounds that repress DUX4 expression in facioscapulohumeral muscular dystrophy.bioRxiv : the preprint server for biology · 2026Article
- CRISPRa-mediated disentanglement of the Dux-MERVL axis in the 2C-like state, totipotency, and cell death.Science advances · 2025Article
- All-in-one vectors for epigenetic CRISPR inhibition ofMolecular therapy. Methods & clinical development · 2025Article
- A discrete region of the D4Z4 is sufficient to initiate epigenetic silencing.Human molecular genetics · 2025Article
- The recent clinical trial of losmapimod for the treatment of facioscapulohumeral muscular dystrophy.Neuromuscular disorders : NMD · 2025Review
- Three-dimensional tissue engineered skeletal muscle modelling facioscapulohumeral muscular dystrophy.Brain : a journal of neurology · 2025Article
- The participants' perspective on facioscapulohumeral muscular dystrophy trials in The Netherlands - A qualitative study.Journal of neuromuscular diseases · 2025Article
- Facioscapulohumeral Dystrophy: Molecular Basis and Therapeutic Opportunities.Cold Spring Harbor perspectives in biology · 2025Review
- Deciphering Facioscapulohumeral Dystrophy in the clinical trials era: where are we now?Acta myologica : myopathies and cardiomyopathies : official journal of the Mediterranean Society of Myology · 2025Review
- Muscular Dystrophies.Advances in experimental medicine and biology · 2025Review
- SIX transcription factors are necessary for the activation of DUX4 expression in facioscapulohumeral muscular dystrophy.Skeletal muscle · 2024Article
- Temporal variation in p38-mediated regulation of DUX4 in facioscapulohumeral muscular dystrophy.Scientific reports · 2024Article
- Molecular mechanisms and therapeutic strategies for neuromuscular diseases.Cellular and molecular life sciences : CMLS · 2024Review
- Flavones provide resistance to DUX4-induced toxicity via an mTor-independent mechanism.Cell death & disease · 2023Article
- Pharmacotherapeutic Approaches to Treatment of Muscular Dystrophies.Biomolecules · 2023Review
- Apabetalone, a Clinical-Stage, Selective BET Inhibitor, Opposes DUX4 Target Gene Expression in Primary Human FSHD Muscle Cells.Biomedicines · 2023Article
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Authors and funding
10 authors at 4 institutions in 1 country.
Jonathan OlivaDepartments of Biochemistry and Molecular Biology (J.O., A.R., N.M., F.M.S.) and Chemistry (M.J.M.), Saint Louis University, St. Louis, Missouri; Ultragenyx Pharmaceutical Inc., Novato, California (S.G.); Human Biology Division, Fred Hutchinson Cancer Research Center, Seattle, Washington (A.E.C., J.W.Z., S.J.T.); Department of Neurology, University of Rochester Medical Center, Rochester, New York (R.T.); and Department of Neurology, University of Washington, Seattle, Washington (S.J.T.).
Scott GalasinskiDepartments of Biochemistry and Molecular Biology (J.O., A.R., N.M., F.M.S.) and Chemistry (M.J.M.), Saint Louis University, St. Louis, Missouri; Ultragenyx Pharmaceutical Inc., Novato, California (S.G.); Human Biology Division, Fred Hutchinson Cancer Research Center, Seattle, Washington (A.E.C., J.W.Z., S.J.T.); Department of Neurology, University of Rochester Medical Center, Rochester, New York (R.T.); and Department of Neurology, University of Washington, Seattle, Washington (S.J.T.).
Amelia RicheyDepartments of Biochemistry and Molecular Biology (J.O., A.R., N.M., F.M.S.) and Chemistry (M.J.M.), Saint Louis University, St. Louis, Missouri; Ultragenyx Pharmaceutical Inc., Novato, California (S.G.); Human Biology Division, Fred Hutchinson Cancer Research Center, Seattle, Washington (A.E.C., J.W.Z., S.J.T.); Department of Neurology, University of Rochester Medical Center, Rochester, New York (R.T.); and Department of Neurology, University of Washington, Seattle, Washington (S.J.T.).
Amy E CampbellDepartments of Biochemistry and Molecular Biology (J.O., A.R., N.M., F.M.S.) and Chemistry (M.J.M.), Saint Louis University, St. Louis, Missouri; Ultragenyx Pharmaceutical Inc., Novato, California (S.G.); Human Biology Division, Fred Hutchinson Cancer Research Center, Seattle, Washington (A.E.C., J.W.Z., S.J.T.); Department of Neurology, University of Rochester Medical Center, Rochester, New York (R.T.); and Department of Neurology, University of Washington, Seattle, Washington (S.J.T.).
Marvin J MeyersDepartments of Biochemistry and Molecular Biology (J.O., A.R., N.M., F.M.S.) and Chemistry (M.J.M.), Saint Louis University, St. Louis, Missouri; Ultragenyx Pharmaceutical Inc., Novato, California (S.G.); Human Biology Division, Fred Hutchinson Cancer Research Center, Seattle, Washington (A.E.C., J.W.Z., S.J.T.); Department of Neurology, University of Rochester Medical Center, Rochester, New York (R.T.); and Department of Neurology, University of Washington, Seattle, Washington (S.J.T.).
Neal ModiDepartments of Biochemistry and Molecular Biology (J.O., A.R., N.M., F.M.S.) and Chemistry (M.J.M.), Saint Louis University, St. Louis, Missouri; Ultragenyx Pharmaceutical Inc., Novato, California (S.G.); Human Biology Division, Fred Hutchinson Cancer Research Center, Seattle, Washington (A.E.C., J.W.Z., S.J.T.); Department of Neurology, University of Rochester Medical Center, Rochester, New York (R.T.); and Department of Neurology, University of Washington, Seattle, Washington (S.J.T.).
Jun Wen ZhongDepartments of Biochemistry and Molecular Biology (J.O., A.R., N.M., F.M.S.) and Chemistry (M.J.M.), Saint Louis University, St. Louis, Missouri; Ultragenyx Pharmaceutical Inc., Novato, California (S.G.); Human Biology Division, Fred Hutchinson Cancer Research Center, Seattle, Washington (A.E.C., J.W.Z., S.J.T.); Department of Neurology, University of Rochester Medical Center, Rochester, New York (R.T.); and Department of Neurology, University of Washington, Seattle, Washington (S.J.T.).
Rabi TawilDepartments of Biochemistry and Molecular Biology (J.O., A.R., N.M., F.M.S.) and Chemistry (M.J.M.), Saint Louis University, St. Louis, Missouri; Ultragenyx Pharmaceutical Inc., Novato, California (S.G.); Human Biology Division, Fred Hutchinson Cancer Research Center, Seattle, Washington (A.E.C., J.W.Z., S.J.T.); Department of Neurology, University of Rochester Medical Center, Rochester, New York (R.T.); and Department of Neurology, University of Washington, Seattle, Washington (S.J.T.).
Stephen J TapscottDepartments of Biochemistry and Molecular Biology (J.O., A.R., N.M., F.M.S.) and Chemistry (M.J.M.), Saint Louis University, St. Louis, Missouri; Ultragenyx Pharmaceutical Inc., Novato, California (S.G.); Human Biology Division, Fred Hutchinson Cancer Research Center, Seattle, Washington (A.E.C., J.W.Z., S.J.T.); Department of Neurology, University of Rochester Medical Center, Rochester, New York (R.T.); and Department of Neurology, University of Washington, Seattle, Washington (S.J.T.).
Francis M SverdrupDepartments of Biochemistry and Molecular Biology (J.O., A.R., N.M., F.M.S.) and Chemistry (M.J.M.), Saint Louis University, St. Louis, Missouri; Ultragenyx Pharmaceutical Inc., Novato, California (S.G.); Human Biology Division, Fred Hutchinson Cancer Research Center, Seattle, Washington (A.E.C., J.W.Z., S.J.T.); Department of Neurology, University of Rochester Medical Center, Rochester, New York (R.T.); and Department of Neurology, University of Washington, Seattle, Washington (S.J.T.) fran.sverdrup@health.slu.edu.
Fred Hutch Cancer Center · USSaint Louis University · USUniversity of Rochester Medical Center · USUniversity of Washington · US
Funding
The Pathogenesis of Facioscapulohumeral Muscular DystrophyP01NS069539 · NINDS · FRED HUTCHINSON CANCER RESEARCH CENTER · PI TAWIL, RABI · 2010 to 2019
$12.3MMYOTONIC DYSTROPHY LOCUS CONTROLR01AR045203 · NIAMS · FRED HUTCHINSON CANCER RESEARCH CENTER · PI TAPSCOTT, STEPHEN J · 1998 to 2025
$9.4MNIAMS NIH HHS R01 AR045203NINDS NIH HHS P01 NS069539
Abstract
Facioscapulohumeral muscular dystrophy (FSHD) is characterized by misexpression of the double homeobox 4 (DUX4) developmental transcription factor in mature skeletal muscle, where it is responsible for muscle degeneration. Preventing expression of DUX4 mRNA is a disease-modifying therapeutic strategy with the potential to halt or reverse the course of disease. We previously reported that agonists of the
Indexed as
AnimalsCell LineDisease Models, AnimalGene Expression RegulationHomeodomain ProteinsMiceMuscle Fibers, SkeletalMuscular Dystrophy, FacioscapulohumeralMyoblastsp38 Mitogen-Activated Protein KinasesProtein Kinase InhibitorsDux4 protein, mouseHomeodomain Proteinsp38 Mitogen-Activated Protein KinasesProtein Kinase Inhibitors
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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.