Casimersen: 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

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• Timing matters: Exon skipping therapy is most effective when initiated early in a mouse model of Duchenne muscular dystrophy

  2026 Molecular Therapy — Nucleic Acids article Biochemistry, Genetics and Molecular Biology Muscle Physiology and Disorders Open access

  0

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• Timing matters: exon skipping therapy is most effective when initiated early in a mouse model of Duchenne muscular dystrophy

  2025 bioRxiv (Cold Spring Harbor Laboratory) preprint Biochemistry, Genetics and Molecular Biology Muscle Physiology and Disorders Open access

  0

  citations
• Understanding Duchenne muscular dystrophy-associated brain pathology

  2025 Disease Models & Mechanisms article Biochemistry, Genetics and Molecular Biology Muscle Physiology and Disorders Open access

  2

  citations
• Non‐coding RNAs to treat vascular smooth muscle cell dysfunction

  2024 British Journal of Pharmacology article Biochemistry, Genetics and Molecular Biology MicroRNA in disease regulation Open access

  15

  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
• Next Generation Exon 51 Skipping Antisense Oligonucleotides for Duchenne Muscular Dystrophy

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

  19

  citations
• The Dilemma of Choice for Duchenne Patients Eligible for Exon 51 Skipping The European Experience

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

  15

  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
• From Antisense RNA to RNA Modification: Therapeutic Potential of RNA-Based Technologies

  2021 Biomedicines article Biochemistry, Genetics and Molecular Biology RNA Interference and Gene Delivery Open access cited by 3 patents

  81

  citations

12 results

• Duchenne muscular dystrophy

  2021 Nature Reviews Disease Primers review Biochemistry, Genetics and Molecular Biology Muscle Physiology and Disorders Open access cited by 15 patents

  1,314

  citations

1 result