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Publications by Annemieke Aartsma-Rus
Development of a Web Course on Gene Therapy by the International Consortium of Gene Therapy
Molecular Therapy
Molecular Medicine
Molecular Biology
Pharmacology
Medicine
Genetics
Drug Discovery
Dystrophin Deficiency Leads to Dysfunctional Glutamate Clearance in iPSC Derived Astrocytes
Translational Psychiatry
Psychiatry
Molecular Neuroscience
Biological Psychiatry
Mental Health
Cellular
RD-Connect, NeurOmics and EURenOmics: Collaborative European Initiative for Rare Diseases
European Journal of Human Genetics
Genetics
Prednisolone Treatment Does Not Interfere With 2′-O-Methyl Phosphorothioate Antisense-Mediated Exon Skipping in Duchenne Muscular Dystrophy
Human Gene Therapy
Molecular Medicine
Genetics
Molecular Biology
Meeting Report of the “Regulatory Exchange Matters” Session at the 5th International TREAT-NMD Conference. Lessons in Communication. How an Early Dialogue Between Patients, Regulators and Academics Can Further Therapy Development for Neuromuscular Disorders.
Neuromuscular Disorders
Child Health
Neurology
Pediatrics
Perinatology
Genetics
Dual Exon Skipping in Myostatin and Dystrophin for Duchenne Muscular Dystrophy
BMC Medical Genomics
Genetics
A Multicenter Comparison of Quantification Methods for Antisense Oligonucleotide-Induced DMD Exon 51 Skipping in Duchenne Muscular Dystrophy Cell Cultures
PLoS ONE
Multidisciplinary
Exon Skipping: A First in Class Strategy for Duchenne Muscular Dystrophy
Expert Opinion on Biological Therapy
Drug Discovery
Clinical Biochemistry
Pharmacology
