Discovery and mechanism of action studies of 4,6-diphenylpyrimidine-2-carbohydrazides as utrophin modulators for the treatment of Duchenne muscular dystrophy.
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Vuorinen A. et al, (2021), Eur J Med Chem, 220
Evaluating the potential of novel genetic approaches for the treatment of Duchenne muscular dystrophy.
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Himič V. and Davies KE., (2021), Eur J Hum Genet
Highway to HHGE: An Interview with Dame Kay E. Davies.
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Davies K. and Davies KE., (2020), CRISPR J, 3, 325 - 331
From diagnosis to therapy in Duchenne muscular dystrophy.
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Babbs A. et al, (2020), Biochem Soc Trans
Isolation, Structural Identification, Synthesis, and Pharmacological Profiling of 1,2-trans-Dihydro-1,2-diol Metabolites of the Utrophin Modulator Ezutromid.
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Chatzopoulou M. et al, (2020), J Med Chem, 63, 2547 - 2556
The Long Journey from Diagnosis to Therapy.
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Davies KE., (2020), Annu Rev Genomics Hum Genet
Publisher Correction: Therapies for rare diseases: therapeutic modalities, progress and challenges ahead.
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Tambuyzer E. et al, (2020), Nat Rev Drug Discov
Chemical Proteomics and Phenotypic Profiling Identifies the Aryl Hydrocarbon Receptor as a Molecular Target of the Utrophin Modulator Ezutromid.
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Wilkinson IVL. et al, (2019), Angew Chem Int Ed Engl
Neuronal over-expression of Oxr1 is protective against ALS-associated mutant TDP-43 mislocalisation in motor neurons and neuromuscular defects in vivo.
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Williamson MG. et al, (2019), Hum Mol Genet, 28, 3584 - 3599
Surrogate gene therapy for muscular dystrophy.
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Davies KE. and Chamberlain JS., (2019), Nat Med, 25, 1473 - 1474
Regenerative biomarkers for Duchenne muscular dystrophy
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Guiraud S. and Davies KE., (2019), Neural Regeneration Research, 14, 1317 - 1320
Limitations to adaptive homeostasis in an hyperoxia-induced model of accelerated ageing.
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Pomatto LCD. et al, (2019), Redox Biol, 24
The potential of utrophin and dystrophin combination therapies for Duchenne muscular dystrophy.
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Guiraud S. et al, (2019), Hum Mol Genet
Micro-dystrophin Genes Bring Hope of an Effective Therapy for Duchenne Muscular Dystrophy.
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Davies KE. and Guiraud S., (2019), Mol Ther
A Phase 1b Trial to Assess the Pharmacokinetics of Ezutromid in Pediatric Duchenne Muscular Dystrophy Patients on a Balanced Diet.
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Muntoni F. et al, (2019), Clin Pharmacol Drug Dev
Embryonic myosin is a regeneration marker to monitor utrophin-based therapies for DMD.
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Guiraud S. et al, (2019), Hum Mol Genet, 28, 307 - 319
Synthesis of SMT022357 enantiomers and in vivo evaluation in a Duchenne muscular dystrophy mouse model
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Babbs A. et al, (2019), Tetrahedron
Micro-utrophin Improves Cardiac and Skeletal Muscle Function of Severely Affected D2/mdx Mice.
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Kennedy TL. et al, (2018), Mol Ther Methods Clin Dev, 11, 92 - 105
Absent sleep EEG spindle activity in GluA1 (Gria1) knockout mice: relevance to neuropsychiatric disorders.
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Ang G. et al, (2018), Transl Psychiatry, 8
Alternative utrophin mRNAs contribute to phenotypic differences between dystrophin-deficient mice and Duchenne muscular dystrophy.
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Perkins KJ. and Davies KE., (2018), FEBS Lett, 592, 1856 - 1869