Viltolarsen: First Approval

• Evaluating exon skipping in the central nervous system in Duchenne muscular dystrophy using spatial transcriptomics

  2026 iScience article Biochemistry, Genetics and Molecular Biology Muscle Physiology and Disorders Open access

  0

  citations
• Antisense-mediated exon skipping therapy improves neuromuscular junction deficits in a Duchenne muscular dystrophy mouse model

  2025 Neuromuscular Disorders article Biochemistry, Genetics and Molecular Biology Muscle Physiology and Disorders Open access

  1

  citations
• ExonViz: A user friendly application to visualize transcripts and genetic variants

  2024 medRxiv preprint Biochemistry, Genetics and Molecular Biology Genomics and Rare Diseases Open access

  0

  citations
• Nucleic acid degradation as barrier to gene delivery: a guide to understand and overcome nuclease activity

  2023 Chemical Society Reviews review Biochemistry, Genetics and Molecular Biology RNA Interference and Gene Delivery cited by 1 patent

  129

  citations
• Challenges of Assessing Exon 53 Skipping of the Human DMD Transcript with Locked Nucleic Acid-Modified Antisense Oligonucleotides in a Mouse Model for Duchenne Muscular Dystrophy

  2023 Nucleic Acid Therapeutics article Biochemistry, Genetics and Molecular Biology Muscle Physiology and Disorders Open access cited by 1 patent

  16

  citations
• 269th ENMC international workshop: 10 years of clinical trials in Duchenne muscular dystrophy – What have we learned? 9–11 December 2022, Hoofddorp, The Netherlands

  2023 Neuromuscular Disorders article Biochemistry, Genetics and Molecular Biology Muscle Physiology and Disorders Open access

  6

  citations
• Cardiovascular complications of diabetes: role of non-coding RNAs in the crosstalk between immune and cardiovascular systems

  2023 Cardiovascular Diabetology article Biochemistry, Genetics and Molecular Biology MicroRNA in disease regulation Open access

  32

  citations
• Next steps for the optimization of exon therapy for Duchenne muscular dystrophy

  2023 Expert Opinion on Biological Therapy article Biochemistry, Genetics and Molecular Biology Muscle Physiology and Disorders Open access cited by 2 patents

  22

  citations
• Low human dystrophin levels prevent cardiac electrophysiological and structural remodelling in a Duchenne mouse model

  2021 Scientific Reports article Medicine Cardiac electrophysiology and arrhythmias Open access

  15

  citations
• Delivery of oligonucleotide‐based therapeutics: challenges and opportunities

  2021 EMBO Molecular Medicine article Biochemistry, Genetics and Molecular Biology RNA Interference and Gene Delivery Open access cited by 13 patents

  388

  citations
• Detailed genetic and functional analysis of the hDMDdel52/mdx mouse model

  2020 PLoS ONE article Biochemistry, Genetics and Molecular Biology Muscle Physiology and Disorders Open access cited by 1 patent

  30

  citations
• Developments in reading frame restoring therapy approaches for Duchenne muscular dystrophy

  2020 Expert Opinion on Biological Therapy article Biochemistry, Genetics and Molecular Biology Muscle Physiology and Disorders Open access

  25

  citations

12 results