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Givinostat for Becker muscular dystrophy- A randomized, placebo-controlled, double-blind study

Monthly #apaperaday wrap-up: March 2023

Prof. Annemieke Aartsma-Rus is taking on a challenge by reading and commenting on a paper a day. She shares her insights, findings and thoughts via her @oligogirl Twitter account. Each month, a curated selection of the relevant papers for Duchenne and Becker muscular dystrophy are presented by the World Duchenne Organization. See below the overview of March 2023.

Must reads

  • Givinostat for Becker muscular dystrophy: A randomized, placebo-controlled, double-blind study Read more>

Interesting papers

  • Macrophage plasticity in Duchenne muscular dystrophy: a nexus of pathological remodelling with therapeutic implications Read more>
  • Muscle Pathology in Dystrophic Rats and Zebrafish Is Unresponsive to Taurine Treatment, Compared to the mdx Mouse Model for Duchenne Muscular Dystrophy Read more>
  • Longitudinal Assessment of Creatine Kinase, Creatine/Creatinine ratio, and Myostatin as Monitoring Biomarkers in Becker Muscular Dystrophy Read more>
  • Urine titin as a novel biomarker for Duchenne muscular dystrophy Read more>

Additional reading

  • Oligonucleotide Enhancing Compound Increases Tricyclo-DNA Mediated Exon-Skipping Efficacy in the Mdx Mouse Model Read more>
  • Investigating the Impact of Delivery Routes for Exon Skipping Therapies in the CNS of DMD Mouse Models Read more>


We are grateful for prof. Aartsma-Rus for allowing us to share her daily recaps. Follow @oligogirl on Twitter to stay on top of the latest #apaperaday tweets, or subscribe to the WDO Newsletter to receive the monthly must-reads in your inbox.


About professor Annemieke Aartsma-Rus

Prof. Dr. Annemieke Aartsma-Rus is a professor of Translational Genetics at the Department of Human Genetics of the Leiden University Medical Center. Since 2013 she has a visiting professorship at the Institute of Genetic Medicine of Newcastle University (UK).

Her work currently focuses on developing antisense-mediated exon skipping as a therapy for Duchenne muscular dystrophy. In addition, in collaborative efforts she aims to bridge the gap between different stakeholders (patients, academics, regulators and industry) involved in drug development for rare diseases.

In 2013 she was elected a member of the junior section of the Dutch Royal Academy of Sciences (KNAW), which consists of what are considered the top 50 scientists in the Netherlands under 45. From 2015 to 2022, she was selected as the most influential scientist in Duchenne muscular dystrophy by Expertscape.