At WMS 2024 in Prague, Czechia, Jordi Diaz Manera sat down with Virginia Arechavala Gomeza and Pietro Spitali to find out more about their careers, their current work and their hopes for the future of the neuromusclar disorders field. 

This video interview is a recording of that conversation. In it, Virginia and Pietro talk about the current treatments for neuromuscular disorders, advances in gene therapies and what it means for traditional treatments and what is happening in the world of neuromuscular disorder research. 

Virginia and Pietro were both invited speakers for day three of WMS 2024, with presentations on the topic of RNA in NMD: clinical insights, patho-mechanisms and treatments. 

Virginia leads the Nucleic Acid Therapeutics for Rare Diseases group at Biobizkaia HRI. She participated in the development of eteplirsen for DMD and she has led a network of stakeholders in the development of better delivery options for these therapies for target tissues.

Pietro Spitali is an associate professor at the Human Genetics department of the Leiden University Medical Center in the Netherlands. His lab focuses on identification of biomarkers in neuromuscular disorders in pre-clinical and clinical studies. He is also actively researching the basic mechanism of gene expression regulation in muscle by studying RNA binding proteins, pre-mRNA synthesis and gene expression at spatial resolution. He is currently co-coordinator of Fluid Biomarker workgroup of the Duchenne Regulatory Science Consortium (D-RSC) of the Critical Path Institute, member of the TREAT-NMD Neuromuscular Disease Advisory Committee (NMDAC) and founding member of the Netherlands single-cell network.

Published on 23 April 2025.

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