The professor for human genetics and neurology at the University of Miami Miller School of Medicine discussed the trend towards gene-targeted approaches in the neuromuscular disease space.
“These therapies are still very expensive. But I do think [these are the] early days, pioneering days—and in fact, prices will come down dramatically over the years. It might take 10 years until this happens. But at the end of the day, I believe that genetic therapies might actually be a very, very affordable mode of medicine.”
In previous decades, there were attempts to develop approaches that could potentially treat a broad range of neuromuscular diseases (NMDs) with a single therapy. However, many of these attempts were not successful, and in recent years there has been increased interest in therapies targeted at specific genetic subtypes of NMDs.
Stephan Züchner, MD, PhD, a professor for human genetics and neurology at the University of Miami Miller School of Medicine, presented in a session entitled “Large-Scale Data Approaches to NMD Research” at the Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, held in Dallas, Texas, March 19-22, 2023.
In an interview with CGTLive™’s sister publication, NeurologyLive™, Züchner spoke about trends in NMD research and treatment over the past 30 years. He noted that today, the most important aspects for patient care are diagnosis, disease management, and the application of disease-modifying therapies, of which there are currently few available. Although, he pointed out that there is hope for the future in this regard, as there are many clinical trials for new therapies currently in progress.
Züchner highlighted the importance of diagnosis, especially genetic diagnosis, for treating patients and identifying avenues of research for new therapies. He noted that many NMD subtypes are caused by single-gene mutations, and that companies and organizations developing new treatments are responding to this by investigating therapies that target these genes. He also noted that while genetic medicines are currently very expensive, he is optimistic that these types of treatments could become much less expensive in the future.