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WDO Webinar: Gene Therapy in Duchenne Muscular Dystrophy

This November 7, the World Duchenne Organization (WDO) convened a webinar for its members to explore the evolving landscape of gene therapy for Duchenne Muscular Dystrophy (DMD). The recording is now available. This article provides an overview of the insights shared during this webinar, emphasizing the importance of research and safety aspects in a field where many unknowns still persist.

Gene Therapy: How it Works

The event commenced with a welcome address by Elizabeth Vroom, Chair of WDO, setting the tone for an objective exploration of gene therapy in the context of DMD. Prof Annemieke Aartsma-Rus offered an in-depth overview of the mechanisms underpinning gene therapy in DMD. While gene therapy holds promise, its complexities and uncertainties were underscored, addressing the necessity for a more cautious approach in the face of unknowns.

Clinical Aspects of Gene Therapy

The webinar’s focus then shifted to the clinical aspects of gene therapy for DMD. Prof Eugenio Mercuri provided valuable insights into managing serious adverse events (SAEs) associated with gene therapy. The uncertainties and potential risks in clinical settings were discussed. Furthermore he emphasized the need for compliance and monitoring for a more comprehensive understanding of the potential consequences.

This was followed by Prof Francesco Muntoni. He addressed the immune responses observed in gene therapy for DMD. The complexities of immune reactions were dissected, pointing to a need for further research to mitigate potential complications. Thirdly, Dr. Chet Villa highlighted the intricacies of gene therapy and its impact on heart health. This presentation served as a reminder of the multifaceted nature of DMD and the importance of addressing all facets of the disease.

Exploring the Gene Therapy Landscape

Following the overview of clinical aspects, the webinar showcased the latest developments in gene therapy. This part featured updates from the pharmaceutical companies Pfizer, Sarepta, Roche, Genethon, Solid BioSciences and RegenXBio. Their presentations offered insight into ongoing research and development. This part was followed by an interactive panel discussion led by Prof Aartsma-Rus, while also taking questions from the audience.

Access & Reimbursement

The last session on access and reimbursement was drawing a parallel from the experience of treating spinal muscular atrophy (SMA). Mencía de Lemus Belmonte underscored the complexities of providing access to cutting-edge treatments and the challenges of reimbursement.

In conclusion, the WDO webinar on Gene Therapy in Duchenne Muscular Dystrophy provided a platform for the objective and scientific exploration of this evolving field. The event served as a reminder of the complexities, uncertainties, and unknowns inherent to gene therapy for DMD.