929 results on '"Aartsma-Rus, Annemieke"'
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52. Development of tailored splice-switching oligonucleotides for progressive brain disorders in Europe: development, regulation, and implementation considerations
53. On the use of D2.B10-Dmdmdx/J (D2.mdx) Versus C57BL/10ScSn-Dmdmdx/J (mdx) Mouse Models for Preclinical Studies on Duchenne Muscular Dystrophy: A Cautionary Note from Members of the TREAT-NMD Advisory Committee on Therapeutics
54. The IRDiRC Chrysalis Task Force: making rare disease research attractive to companies
55. Measuring clinical effectiveness of medicinal products for the treatment of Duchenne muscular dystrophy
56. Orthogonal proteomics methods warrants development of Duchenne Muscular Dystrophy biomarkers
57. Whole‐genome sequencing holds the key to the success of gene‐targeted therapies
58. Histone deacetylase inhibitors improve antisense-mediated exon-skipping efficacy in mdx mice
59. Good news for the mdx mouse community: Improved dystrophin restoration after skipping mouse dystrophin exon 23
60. Why dystrophin quantification is key in the eteplirsen saga
61. Delivery is key: lessons learnt from developing splice‐switching antisense therapies
62. Mimicking Cardiac Fibrosis in a Dish: Fibroblast Density Rather than Collagen Density Weakens Cardiomyocyte Function
63. DMDantisense oligonucleotide mediated exon skipping efficiency is affected by flanking intron retention time and target position within the exon
64. Antisense-Mediated Exon Skipping for Duchenne Muscular Dystrophy
65. Biomarkers and surrogate endpoints in Duchenne: Meeting report
66. Low dystrophin levels in heart can delay heart failure in mdx mice
67. A Novel Feed-Forward Loop between ARIH2 E3-Ligase and PABPN1 Regulates Aging-Associated Muscle Degeneration
68. Measuring DNA hybridization using fluorescent DNA-stabilized silver clusters to investigate mismatch effects on therapeutic oligonucleotides
69. In-Depth Characterization of Protein Disulfide Bonds by Online Liquid Chromatography-Electrochemistry-Mass Spectrometry
70. CRISPR applications for Duchenne muscular dystrophy: From animal models to potential therapies
71. Sexual Dimorphism in Transcriptional and Functional Glucocorticoid Effects on Mouse Skeletal Muscle
72. 194th ENMC international workshop. 3rd ENMC workshop on exon skipping: Towards clinical application of antisense-mediated exon skipping for Duchenne muscular dystrophy 8–10 December 2012, Naarden, The Netherlands
73. Advances in therapeutic RNA-targeting
74. Sexual Dimorphism in Transcriptional and Functional Glucocorticoid Effects on Mouse Skeletal Muscle
75. Whole‐genome sequencing holds the key to the success of gene‐targeted therapies.
76. Antisense‐mediated exon skipping: a therapeutic strategy for titin‐based dilated cardiomyopathy
77. Comparison of skeletal muscle pathology and motor function of dystrophin and utrophin deficient mouse strains
78. Assessment of cardiac function in three mouse dystrophinopathies by magnetic resonance imaging
79. Letter by Duan et al Regarding Article, “Therapeutic Exon Skipping Through a CRISPR-Guided Cytidine Deaminase Rescues Dystrophic Cardiomyopathy In Vivo”
80. The Role of Patient Involvement When Developing Therapies
81. Spatial transcriptomics reveal markers of histopathological changes in Duchenne muscular dystrophy mouse models
82. The therapeutic potential of soluble activin type receptor IIB treatment in a limb girdle muscular dystrophy type 2D mouse model
83. Orphan Medicine Incentives: How to Address the Unmet Needs of Rare Disease Patients by Optimizing the European Orphan Medicinal Product Landscape Guiding Principles and Policy Proposals by the European Expert Group for Orphan Drug Incentives (OD Expert Group)
84. Affinity proteomics within rare diseases: a BIO‐NMD study for blood biomarkers of muscular dystrophies
85. Therapeutic NOTCH3 cysteine correction in CADASIL using exon skipping: in vitro proof of concept
86. The importance of genetic diagnosis for Duchenne muscular dystrophy
87. Consensus Guidelines for the Design and In Vitro Preclinical Efficacy Testing N-of-1 Exon Skipping Antisense Oligonucleotides.
88. CRISPR applications for Duchenne muscular dystrophy: From animal models to potential therapies.
89. On the use of D2.B10-Dmdmdx/J (D2.mdx) Versus C57BL/10ScSn-Dmdmdx/J (mdx) Mouse Models for Preclinical Studies on Duchenne Muscular Dystrophy: A Cautionary Note from Members of the TREAT-NMD Advisory Committee on Therapeutics.
90. Accurate quantification of dystrophin mRNA and exon skipping levels in Duchenne Muscular Dystrophy
91. Preclinical PK and PD Studies on 2′-O-Methyl-phosphorothioate RNA Antisense Oligonucleotides in the mdx Mouse Model
92. A 3 months mild functional test regime does not affect disease parameters in young mdx mice
93. Comparative analysis of antisense oligonucleotide sequences targeting exon 53 of the human DMD gene: Implications for future clinical trials
94. 7′,5′-alpha-bicyclo-DNA: new chemistry for oligonucleotide exon splicing modulation therapy
95. Sharing “Negative” Results in Neuromuscular Research: A Positive Experience
96. Author’s Response to: Rebuttal to: Simvastatin Treatment Does Not Ameliorate Muscle Pathophysiology in a Mouse Model for Duchenne Muscular Dystrophy, Verhaart et al. 2020
97. Simvastatin Treatment Does Not Ameliorate Muscle Pathophysiology in a Mouse Model for Duchenne Muscular Dystrophy
98. Case Report: The Genetic Diagnosis of Duchenne Muscular Dystrophy in the Middle East
99. DMDGenotypes and Motor Function in Duchenne Muscular Dystrophy
100. Validation of genetic modifiers for Duchenne muscular dystrophy: a multicentre study assessing SPP1 and LTBP4 variants
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