Therapeutic developments for Duchenne muscular dystrophy

• 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
• The Dutch Dystrophinopathy Database: A National Registry with Standardized Patient and Clinician Reported Real-World Data

  2024 Journal of Neuromuscular Diseases article Biochemistry, Genetics and Molecular Biology Muscle Physiology and Disorders Open access

  7

  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
• Neuromuscular disease genetics in under-represented populations: increasing data diversity

  2023 Brain article Biochemistry, Genetics and Molecular Biology Genomics and Rare Diseases Open access

  31

  citations
• Efficient Downregulation of Alk4 in Skeletal Muscle After Systemic Treatment with Conjugated siRNAs in a Mouse Model for Duchenne Muscular Dystrophy

  2022 Nucleic Acid Therapeutics article Biochemistry, Genetics and Molecular Biology RNA Interference and Gene Delivery Open access

  14

  citations
• The Black Box of Technological Outcome Measures: An Example in Duchenne Muscular Dystrophy

  2022 Journal of Neuromuscular Diseases article Medicine Neurogenetic and Muscular Disorders Research Open access

  8

  citations
• Genotype‐related respiratory progression in Duchenne muscular dystrophy—A multicenter international study

  2021 Muscle & Nerve article Biochemistry, Genetics and Molecular Biology Muscle Physiology and Disorders Open access

  11

  citations
• Association of Elbow Flexor MRI Fat Fraction With Loss of Hand-to-Mouth Movement in Patients With Duchenne Muscular Dystrophy

  2021 Neurology article Biochemistry, Genetics and Molecular Biology Muscle Physiology and Disorders Open access

  12

  citations
• Preserved thenar muscles in non‐ambulant Duchenne muscular dystrophy patients

  2021 Journal of Cachexia Sarcopenia and Muscle article Biochemistry, Genetics and Molecular Biology Muscle Physiology and Disorders Open access

  9

  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
• Peripheral blood transcriptome profiling enables monitoring disease progression in dystrophic mice and patients

  2021 EMBO Molecular Medicine article Biochemistry, Genetics and Molecular Biology Muscle Physiology and Disorders Open access

  23

  citations
• 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
• 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
• Simvastatin Treatment Does Not Ameliorate Muscle Pathophysiology in a Mouse Model for Duchenne Muscular Dystrophy

  2020 Journal of Neuromuscular Diseases article Biochemistry, Genetics and Molecular Biology Muscle Physiology and Disorders Open access

  17

  citations
• Genetic testing offer for inherited neuromuscular diseases within the EURO-NMD reference network: A European survey study

  2020 PLoS ONE article Biochemistry, Genetics and Molecular Biology Genomics and Rare Diseases Open access

  11

  citations
• Decision-Making And Selection Bias in Four Observational Studies on Duchenne and Becker Muscular Dystrophy

  2020 Journal of Neuromuscular Diseases article Medicine Ethics in Clinical Research Open access

  14

  citations
• Safety issues and harmful pharmacological interactions of nutritional supplements in Duchenne muscular dystrophy: considerations for Standard of Care and emerging virus outbreaks

  2020 Pharmacological Research article Medicine Complementary and Alternative Medicine Studies Open access

  19

  citations
• High-Capacity Adenoviral Vectors Permit Robust and Versatile Testing of DMD Gene Repair Tools and Strategies in Human Cells

  2020 Cells article Biochemistry, Genetics and Molecular Biology Virus-based gene therapy research Open access

  30

  citations
• Mouse models for muscular dystrophies: an overview

  2020 Disease Models & Mechanisms article Biochemistry, Genetics and Molecular Biology Muscle Physiology and Disorders Open access

  66

  citations
• MRI vastus lateralis fat fraction predicts loss of ambulation in Duchenne muscular dystrophy

  2020 Neurology article Biochemistry, Genetics and Molecular Biology Muscle Physiology and Disorders Open access

  95

  citations

20 results

• Safety issues and harmful pharmacological interactions of nutritional supplements in Duchenne muscular dystrophy: considerations for Standard of Care and emerging virus outbreaks

  2020 Pharmacological Research article Medicine Complementary and Alternative Medicine Studies Open access

  19

  citations

1 result