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Annemieke Aartsma-Rus
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1

Possibilities and limitations of antisense oligonucleotide ..:

Lauffer, Marlen C. ; van Roon-Mom, Willeke ; Aartsma-Rus, Annemieke.
Communications Medicine.  4 (2024)  1 - p. , 2024
Link: https://doi.org/10.1038/..
 
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2

Treatability of the KMT2-Associated Neurodevelopmental Diso..:

Zardetto, Bianca ; van Roon-Mom, Willeke ; Aartsma-Rus, Annemieke..
Human Mutation.  2024 (2024)  - p. 1-17 , 2024
Link: https://doi.org/10.1155/..
 
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3

P171: Developing an approach to screening rare genetic diag..:

Cheerie, David ; Lauffer, Marlen ; Beijer, Danique...
Genetics in Medicine Open.  2 (2024)  - p. 101068 , 2024
Link: https://doi.org/10.1016/..
 
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4

Antisense Oligonucleotide-Mediated Downregulation of IGFBPs..:

Yavas, Alper ; van Putten, Maaike ; Aartsma-Rus, Annemieke
Journal of Neuromuscular Diseases.  11 (2024)  2 - p. 299-314 , 2024
Link: https://doi.org/10.3233/..
 
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5

The IRDiRC Chrysalis Task Force: making rare disease resear..:

Beaverson, Katherine L. ; Julkowska, Daria ; Letinturier, Mary Catherine V....
Therapeutic Advances in Rare Disease.  4 (2023)  - p. , 2023
Link: https://doi.org/10.1177/..
 
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6

DMDGenotypes and Motor Function in Duchenne Muscular Dystro..:

Muntoni, Francesco ; Signorovitch, James ; Sajeev, Gautam...
Neurology.  100 (2023)  15 - p. , 2023
Link: https://doi.org/10.1212/..
 
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7

Orthogonal proteomics methods warrant the development of Du..:

Johansson, Camilla ; Hunt, Helian ; Signorelli, Mirko...
Clinical Proteomics.  20 (2023)  1 - p. , 2023
Link: https://doi.org/10.1186/..
 
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8

DMDantisense oligonucleotide mediated exon skipping efficie..:

Goossens, Remko ; Verwey, Nisha ; Ariyurek, Yavuz..
RNA Biology.  20 (2023)  1 - p. 693-702 , 2023
Link: https://doi.org/10.1080/..
 
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9

The Dilemma of Choice for Duchenne Patients Eligible for Ex..:

Aartsma-Rus, Annemieke ; De Waele, Liesbeth ; Houwen-Opstal, Saskia...
Journal of Neuromuscular Diseases.  10 (2023)  3 - p. 315-325 , 2023
Link: https://doi.org/10.3233/..
 
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10

Lessons learned from the first national population-based ge..:

Singer, Amihood ; Aartsma-Rus, Annemieke ; Grinshpun-Cohen, Julia.
Genetics in Medicine.  25 (2023)  12 - p. 100981 , 2023
Link: https://doi.org/10.1016/..
 
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11

Multiomic characterization of disease progression in mice l..:

Signorelli, Mirko ; Tsonaka, Roula ; Aartsma-Rus, Annemieke..
PLOS ONE.  18 (2023)  3 - p. e0283869 , 2023
Link: https://doi.org/10.1371/..
 
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12

Gene therapy for X-linked myotubular myopathy: the challeng..:

Voermans, Nicol C ; Ferreiro, Ana ; Aartsma-Rus, Annemieke.
The Lancet Neurology.  22 (2023)  12 - p. 1089-1091 , 2023
Link: https://doi.org/10.1016/..
 
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13

Next steps for the optimization of exon therapy for Duchenn..:

Filonova, Galina ; Aartsma-Rus, Annemieke
Expert Opinion on Biological Therapy.  23 (2023)  2 - p. 133-143 , 2023
Link: https://doi.org/10.1080/..
 
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14

Good news for the mdx mouse community: Improved dystrophin ..:

Aartsma-Rus, Annemieke
Molecular Therapy - Nucleic Acids.  30 (2022)  - p. 355-356 , 2022
Link: https://doi.org/10.1016/..
 
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15

Sexual Dimorphism in Transcriptional and Functional Glucoco..:

Li, Sheng ; Schönke, Milena ; Buurstede, Jacobus C....
Frontiers in Endocrinology.  13 (2022)  - p. , 2022
Link: https://doi.org/10.3389/..
 
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