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Publications

Discovery and mechanism of action studies of 4,6-diphenylpyrimidine-2-carbohydrazides as utrophin modulators for the treatment of Duchenne muscular dystrophy.

Vuorinen A. et al, (2021), Eur J Med Chem, 220

Evaluating the potential of novel genetic approaches for the treatment of Duchenne muscular dystrophy.

Himič V. and Davies KE., (2021), Eur J Hum Genet

Highway to HHGE: An Interview with Dame Kay E. Davies.

Davies K. and Davies KE., (2020), CRISPR J, 3, 325 - 331

From diagnosis to therapy in Duchenne muscular dystrophy.

Babbs A. et al, (2020), Biochem Soc Trans

The Long Journey from Diagnosis to Therapy.

Davies KE., (2020), Annu Rev Genomics Hum Genet

Publisher Correction: Therapies for rare diseases: therapeutic modalities, progress and challenges ahead.

Tambuyzer E. et al, (2020), Nat Rev Drug Discov

Therapies for rare diseases: therapeutic modalities, progress and challenges ahead.

Tambuyzer E. et al, (2019), Nature reviews. Drug discovery

Chemical Proteomics and Phenotypic Profiling Identifies the Aryl Hydrocarbon Receptor as a Molecular Target of the Utrophin Modulator Ezutromid.

Wilkinson IVL. et al, (2019), Angew Chem Int Ed Engl

Isolation, structural identification, synthesis, and pharmacological profiling of 1,2-trans-dihydro-1,2-diol metabolites of the utrophin modulator ezutromid

Chatzopoulou M. et al, (2019), Journal of Medicinal Chemistry, 63, 2547 - 2556

Surrogate gene therapy for muscular dystrophy

Davies KE. and Chamberlain JS., (2019), Nature Medicine, 25, 1473 - 1474

Neuronal over-expression of OXR1 is protective against ALS-associated mutant TDP-43 mislocalisation in motor neurons and neuromuscular defects in vivo

Williamson MG. et al, (2019), Human Molecular Genetics

The potential of utrophin and dystrophin combination therapies for Duchenne muscular dystrophy.

Guiraud S. et al, (2019), Hum Mol Genet, 28, 2189 - 2200

Limitations to adaptive homeostasis in an hyperoxia-induced model of accelerated ageing.

Pomatto LCD. et al, (2019), Redox Biol, 24

Regenerative biomarkers for Duchenne muscular dystrophy

Guiraud S. and Davies KE., (2019), Neural Regeneration Research, 14, 1317 - 1320

Micro-dystrophin Genes Bring Hope of an Effective Therapy for Duchenne Muscular Dystrophy

Davies KE. and Guiraud S., (2019), Molecular therapy : the journal of the American Society of Gene Therapy, 27, 486 - 488

A Phase 1b Trial to Assess the Pharmacokinetics of Ezutromid in Pediatric Duchenne Muscular Dystrophy Patients on a Balanced Diet.

Muntoni F. et al, (2019), Clin Pharmacol Drug Dev

Embryonic myosin is a regeneration marker to monitor utrophin-based therapies for DMD.

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

Babbs A. et al, (2019), Tetrahedron

Micro-utrophin Improves Cardiac and Skeletal Muscle Function of Severely Affected D2/mdx Mice.

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

Vyazovskiy V. et al, (2018), Translational Psychiatry

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