| 1 |
Molecular Heterogeneity and Response to Neoadjuvant Human Epidermal Growth Factor Receptor 2 Targeting in CALGB 40601, a Randomized Phase III Trial of Paclitaxel Plus Trastuzumab With or Without Lapatinib
Lisa A. Carey, Donald A. Berry, Constance Cirrincione et al. · Journal of Clinical Oncology
Open access |
2 Nov 2015 |
442 |
| 2 |
Benchmarking of commercially available CHO cell culture media for antibody production
David Reinhart, Lukas Damjanovic, Christian Kaisermayer et al. · Applied Microbiology and Biotechnology
Open access |
5 Apr 2015 |
146 |
| 3 |
Use of capillary Western immunoassay (Wes) for quantification of dystrophin levels in skeletal muscle of healthy controls and individuals with Becker and Duchenne muscular dystrophy
Chantal Beekman, Anneke A. M. Janson, Aabed Baghat et al. · PLoS ONE
Open access |
11 Apr 2018 |
136 |
| 4 |
Long-Term Efficacy, Safety, and Pharmacokinetics of Drisapersen in Duchenne Muscular Dystrophy: Results from an Open-Label Extension Study
Nathalie Goemans, M. Tulinius, M. van den Hauwe et al. · PLoS ONE
Open access |
2 Sep 2016 |
94 |
| 5 |
The expanded CAG repeat in the huntingtin gene as target for therapeutic RNA modulation throughout the HD mouse brain
Nicole A. Datson, Anchel González‐Barriga, Eleni Kourkouta et al. · PLoS ONE
Open access |
9 Feb 2017 |
74 |
| 6 |
Suppression of Mutant Protein Expression in SCA3 and SCA1 Mice Using a CAG Repeat-Targeting Antisense Oligonucleotide
Eleni Kourkouta, Rudie Weij, Anchel González‐Barriga et al. · Molecular Therapy — Nucleic Acids
Open access |
20 Jul 2019 |
65 |
| 7 |
Report of a TREAT-NMD/World Duchenne Organisation Meeting on Dystrophin Quantification Methodology
Annemieke M Aartsma-Rus, on behalf of the workshop participants, Jennifer Elizabeth Morgan et al. · Journal of Neuromuscular Diseases
Open access |
4 Jan 2019 |
57 |
| 8 |
Digital Droplet PCR for the Absolute Quantification of Exon Skipping Induced by Antisense Oligonucleotides in (Pre-)Clinical Development for Duchenne Muscular Dystrophy
Ruurd C. Verheul, J.C.T. van Deutekom, Nicole A. Datson · PLoS ONE
Open access |
9 Sep 2016 |
39 |
| 9 |
Placebo‐controlled Phase 2 Trial of Drisapersen for Duchenne Muscular Dystrophy
Craig M. McDonald, Brenda L. Y. Wong, Kevin M. Flanigan et al. · Annals of Clinical and Translational Neurology
Open access |
11 Jul 2018 |
37 |
| 10 |
Evaluation of serum MMP-9 as predictive biomarker for antisense therapy in Duchenne
Afrodite Lourbakos et al. · Scientific Reports
Open access |
14 Dec 2017 |
31 |
| 11 |
Cyclic Peptides to Improve Delivery and Exon Skipping of Antisense Oligonucleotides in a Mouse Model for Duchenne Muscular Dystrophy
Silvana M.G. Jirka, Peter A.C. ’t Hoen, Valeriano Diaz Parillas et al. · Molecular Therapy
Open access |
13 Oct 2017 |
31 |
| 12 |
Detailed genetic and functional analysis of the hDMDdel52/mdx mouse model
Alper Yavas, Rudie Weij, Maaike van Putten et al. · PLoS ONE
Open access |
23 Dec 2020 |
30 |
| 13 |
Intracellular Distribution and Nuclear Activity of Antisense Oligonucleotides After Unassisted Uptake in Myoblasts and Differentiated Myotubes In Vitro
Anchel González‐Barriga, Bram Nillessen, Julia Kranzen et al. · Nucleic Acid Therapeutics
Open access |
4 Apr 2017 |
30 |
| 14 |
The Pharmacokinetics of 2′- O -Methyl Phosphorothioate Antisense Oligonucleotides: Experiences from Developing Exon Skipping Therapies for Duchenne Muscular Dystrophy
Sieto Bosgra, Jessica A. Sipkens, Sjef de Kimpe et al. · Nucleic Acid Therapeutics
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20 Aug 2019 |
29 |
| 15 |
A multicenter comparison of quantification methods for antisense oligonucleotide-induced DMD exon 51 skipping in Duchenne muscular dystrophy cell cultures
Monika Hiller, Maria Sofia Falzarano, Iker García et al. · PLoS ONE
Open access |
2 Oct 2018 |
28 |
| 16 |
Low dystrophin variability between muscles and stable expression over time in Becker muscular dystrophy using capillary Western immunoassay
Zaïda Koeks, Annika Janson, Chantal Beekman et al. · Scientific Reports
Open access |
15 Mar 2021 |
24 |
| 17 |
Therapy with 2′-O-Me Phosphorothioate Antisense Oligonucleotides Causes Reversible Proteinuria by Inhibiting Renal Protein Reabsorption
Manoe Jacoba Janssen, Tom T.G. Nieskens, Tessa A. M. Steevels et al. · Molecular Therapy — Nucleic Acids
Open access |
6 Sep 2019 |
23 |
| 18 |
Assessment of Dermal Absorption of Aluminum from a Representative Antiperspirant Formulation Using a 26Al Microtracer Approach
Rianne de Ligt, Esther van Duijn, Dimitri Grossouw et al. · Clinical and Translational Science
Open access |
27 Jul 2018 |
23 |
| 19 |
Evaluation of 2’-Deoxy-2’-fluoro Antisense Oligonucleotides for Exon Skipping in Duchenne Muscular Dystrophy
Silvana M.G. Jirka, Christa L. Tanganyika-de Winter, Joke W. Boertje-van der Meulen et al. · Molecular Therapy — Nucleic Acids
Open access |
2015 |
23 |
| 20 |
The licorice pentacyclic triterpenoid component 18β-glycyrrhetinic acid enhances the activity of antibiotics against strains of methicillin-resistant Staphylococcus aureus
Anna de Breij, T. G. Karnaoukh, Jasmijn A. Schrumpf et al. · European Journal of Clinical Microbiology & Infectious Diseases
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15 Jan 2016 |
21 |
| 21 |
Next Generation Exon 51 Skipping Antisense Oligonucleotides for Duchenne Muscular Dystrophy
Judith Van Deutekom et al. · Nucleic Acid Therapeutics
Open access |
10 Apr 2023 |
20 |
| 22 |
Comparison of ambulatory capacity and disease progression of Duchenne muscular dystrophy subjects enrolled in the drisapersen DMD114673 study with a matched natural history cohort of subjects on daily corticosteroids
Nathalie Goemans, M. Tulinius, Anna‐Karin Kroksmark et al. · Neuromuscular Disorders
Open access |
25 Nov 2016 |
20 |
| 23 |
Fingerprinting of neurotoxic compounds using a mouse embryonic stem cell dual luminescence reporter assay
Marilena Colaianna, Sten Ilmjärv, Hedi Peterson et al. · Archives of Toxicology
Open access |
25 Mar 2016 |
16 |
| 24 |
Pediatric Extrapolation in Type 2 Diabetes: Future Implications of a Workshop
Jeffrey S. Barrett, Christina M. Bucci-Rechtweg, S. Y. Amy Cheung et al. · Clinical Pharmacology & Therapeutics
Open access |
4 Feb 2020 |
13 |
| 25 |
Using a State-of-the-Art Toolbox to Evaluate Molecular and Functional Readouts of Antisense Oligonucleotide-Induced Exon Skipping in mdx Mice
Nicole A. Datson, Suzanne Bijl, Anneke A. M. Janson et al. · Nucleic Acid Therapeutics
Open access |
10 Dec 2019 |
11 |
| 26 |
Nonclinical Exon Skipping Studies with 2′- O -Methyl Phosphorothioate Antisense Oligonucleotides in mdx and mdx-utrn−/− Mice Inspired by Clinical Trial Results
Maaike van Putten, C. Tanganyika-de Winter, Sieto Bosgra et al. · Nucleic Acid Therapeutics
Open access |
23 Jan 2019 |
9 |
| 27 |
Persistent Hypersomnolence Following Clobazam in a Child With Epilepsy and Undiagnosed CYP2C19 Polymorphism
Katherine E. Hamilton, Chasity M. Shelton, James W. Wheless et al. · The Journal of Pediatric Pharmacology and Therapeutics
Open access |
2020 |
7 |
| 28 |
Feasibility of SPECT-CT Imaging to Study the Pharmacokinetics of Antisense Oligonucleotides in a Mouse Model of Duchenne Muscular Dystrophy
Evita van de Steeg, Tilman Läppchen, Begoña Aguilera et al. · Nucleic Acid Therapeutics
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18 Apr 2017 |
5 |
| 29 |
Exon 51 Skipping Quantification by Digital Droplet PCR in del52hDMD/mdx Mice
Monika Hiller, Pietro Spitali, Nicole A. Datson et al. · Methods in molecular biology
Book chapter |
2018 |
4 |
| 30 |
How Do Individuals Value Worse-Than-Dead EQ-5D-5L Health States in Composite Time Trade-Off Tasks? A Qualitative Study
Meixia Liao, Zhihao Yang, Milad Karimi et al. · Value in Health
Open access |
6 Feb 2025 |
1 |
| 31 |
Minimum dietary level and mix ratio of krill meal and fish meal to elicit feed intake and growth performance in juvenile Penaeus vannamei
Alberto Jorge Pinto Nunes, Jordana Sampaio Leite, Caio Gabriel Dantas Gomes et al. · Research Square
PreprintOpen access |
12 Feb 2024 |
0 |
| 32 |
L2 The expanded cag repeat in the huntingtin gene as target for therapeutic RNA modulation throughout the R6/2 and Q175 HD mouse brain
Anchel González‐Barriga, Eleni Kourkouta, Outi Kontkanen et al. · Journal of Neurology Neurosurgery & Psychiatry
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Sep 2016 |
0 |
| 33 |
Comparing biomarker expression between sub-populations of Duchenne muscular dystrophy patients, measured using immunostaining in muscle biopsies
Karin Boekhoorn, Chantal Beekman, Simone van Wijngaarden et al. · Neuromuscular Disorders
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9 Sep 2016 |
0 |
| 34 |
Effect of different dosing regimens of exon skipping antisense oligonucleotides on functional outcome in the mdx mouse model of Duchenne muscular dystrophy
Nicole A. Datson, Rudie Weij, Suzanne Bijl et al. · Neuromuscular Disorders
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10 Sep 2015 |
0 |
| 35 |
Longitudinal quantitative muscle MRI in 5 Duchenne boys on and off drisapersen treatment
Melissa T. Hooijmans, Beatrijs H. A. Wokke, Natalie Goemans et al. · Neuromuscular Disorders
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10 Sep 2015 |
0 |
| 36 |
Development of a patient-reported outcome measure for arm and hand function in Duchenne muscular dystrophy (UL-PROM DMD)
Katrijn Klingels, Anna G Mayhew, Elena Stacy Mazzone et al. · Neuromuscular Disorders
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10 Sep 2015 |
0 |