Search results (345)
« Back to PublicationsSynthesis of aryl-fused bicyclo[3.1.1]heptanes and validation as naphthyl bioisosteres
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Kerckhoffs A. et al, (2026), Nature Chemistry
Personal journeys to and in human genetics and dysmorphology.
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Schwartz CE. et al, (2024), American journal of medical genetics. Part A, 194
Corrigendum to "Long-term clinical follow-up of a family with Becker muscular dystrophy associated with a large deletion in the DMD gene" Neuromuscular Disorders 39 (2024) 5-9.
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Davies KE. and Vogt J., (2024), Neuromuscular disorders : NMD, 39
Long-term clinical follow-up of a family with Becker muscular dystrophy associated with a large deletion in the DMD gene.
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Davies KE. and Vogt J., (2024), Neuromuscular disorders : NMD, 39, 5 - 9
Therapeutic approaches for Duchenne muscular dystrophy
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Roberts TC. et al, (2023), Nature Reviews Drug Discovery, 22, 917 - 934
Utrophin correlates with disease severity in Duchenne muscular dystrophy.
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Guiraud S. and Davies K., (2023), Med (New York, N.Y.), 4, 220 - 222
Evaluating the potential of novel genetic approaches for the treatment of Duchenne muscular dystrophy.
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Himič V. and Davies KE., (2021), European journal of human genetics : EJHG, 29, 1369 - 1376
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), European Journal of Medicinal Chemistry, 220, 113431 - 113431
Highway to HHGE: An Interview with Dame Kay E. Davies.
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Davies K. and Davies KE., (2020), The CRISPR journal, 3, 325 - 331
The Long Journey from Diagnosis to Therapy.
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Davies KE., (2020), Annual review of genomics and human genetics, 21, 1 - 13
From diagnosis to therapy in Duchenne muscular dystrophy
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Babbs A. et al, (2020), Biochemical Society Transactions, 48, 813 - 821
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), Journal of Medicinal Chemistry, 63, 2547 - 2556
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, (2020), Angewandte Chemie International Edition, 59, 2420 - 2428
Therapies for rare diseases: therapeutic modalities, progress and challenges ahead.
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Tambuyzer E. et al, (2020), Nature reviews. Drug discovery, 19, 93 - 111
Synthesis of SMT022357 enantiomers and in vivo evaluation in a Duchenne muscular dystrophy mouse model
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Babbs A. et al, (2020), Tetrahedron, 76, 130819 - 130819