Annemieke M. Rus-Aartsma

Central Parkway
Newcastle Upon Tyne, ENG, United Kingdom

Last Updated: 09/26/2026

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Found 242 publications

TREAT-NMD advisory committee for therapeutics: Preclinical and clinical learnings from 15 years of TACT.

TREAT-NMD advisory committee for therapeutics: Preclinical and clinical learnings from 15 years of TACT.

Journal: Journal of neuromuscular diseases

Published: April 24, 2026

Individualized antisense oligonucleotide treatment eligibility of patients living with neurodevelopmental diseases.

Individualized antisense oligonucleotide treatment eligibility of patients living with neurodevelopmental diseases.

Journal: Cell reports. Medicine

Published: March 18, 2026

Conjugated Antisense Oligonucleotides for Skipping of Duchenne Muscular Dystrophy Exon 53: A Cautionary Study.

Conjugated Antisense Oligonucleotides for Skipping of Duchenne Muscular Dystrophy Exon 53: A Cautionary Study.

Journal: Nucleic acid therapeutics

Published: March 12, 2026

Conjugated Antisense Oligonucleotides for Skipping of Duchenne Muscular Dystrophy Exon 53: A Cautionary Study.

Conjugated Antisense Oligonucleotides for Skipping of Duchenne Muscular Dystrophy Exon 53: A Cautionary Study.

Journal: Nucleic acid therapeutics

Published: March 12, 2026

Four new mouse models of Duchenne muscular dystrophy with clinically relevant exon deletions in the human DMD gene.

Four new mouse models of Duchenne muscular dystrophy with clinically relevant exon deletions in the human DMD gene.

Journal: Disease models & mechanisms

Published: February 12, 2026

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

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

Journal: iScience

Published: January 08, 2026

Guidance on communication and informed consent with patients and their families for experimental individualized treatments.

Guidance on communication and informed consent with patients and their families for experimental individualized treatments.

Journal: American journal of human genetics

Published: December 19, 2025

Report of the multistakeholder drug development round table meeting of the World Duchenne Organization focusing on challenges for clinical development of therapies.

Report of the multistakeholder drug development round table meeting of the World Duchenne Organization focusing on challenges for clinical development of therapies.

Journal: Journal of neuromuscular diseases

Published: December 04, 2025

RNA therapies are delivering as therapies for neuromuscular dystrophy patients: Editorial for the special issue on RNA therapies in Journal of Neuromuscular Diseases.

RNA therapies are delivering as therapies for neuromuscular dystrophy patients: Editorial for the special issue on RNA therapies in Journal of Neuromuscular Diseases.

Journal: Journal of neuromuscular diseases

Published: December 04, 2025

Unraveling the spatial landscape of dystrophinopathies: a transcriptomic approach to Becker and Duchenne muscular dystrophies.

Unraveling the spatial landscape of dystrophinopathies: a transcriptomic approach to Becker and Duchenne muscular dystrophies.

Journal: The Journal of pathology

Published: November 10, 2025

Myonuclear Domain-Associated and Central Nucleation-Dependent Spatial Restriction of Dystrophin Protein Expression.

Myonuclear Domain-Associated and Central Nucleation-Dependent Spatial Restriction of Dystrophin Protein Expression.

Journal: Journal of cachexia, sarcopenia and muscle

Published: October 02, 2025

In Vivo Phage Display for the Identification of Muscle Homing Peptides to Improve the Delivery of Phosphorodiamidate Morpholino Oligomers for Duchenne Muscular Dystrophy Therapy.

In Vivo Phage Display for the Identification of Muscle Homing Peptides to Improve the Delivery of Phosphorodiamidate Morpholino Oligomers for Duchenne Muscular Dystrophy Therapy.

Journal: Nucleic acid therapeutics

Published: August 29, 2025

Showing 1-12 of 242

Last Updated: 09/26/2026