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Explore the transformative journey of Percheron Therapeutics with Dr. James Garner at the helm, as he shares the company's pioneering efforts in the field of rare disease therapies. Discover how their strategic evolution from Antisense Therapeutics to Percheron Therapeutics has positioned them at the forefront of developing groundbreaking treatments for Duchenne muscular dystrophy (DMD). As we delve into the intricacies of their phase 2b trial of avicursen for non-ambulant boys, Dr. Garner reveals the innovative use of the Performance in the Upper Limb (PUL2.0) endpoint and the promising results from their essential non-human primate toxicology study, aligning with FDA requirements for future clinical trials.

Uncover the strategic blueprint Percheron employs to bring their therapies to market, leveraging Ionis Pharmaceuticals expertise and carefully outsourcing critical tasks, allowing them to concentrate on clinical development. Dr. Garner outlines their ambitious commercialization plans, aiming to partner with larger entities for broader market reach. With pivotal data readouts expected soon and ongoing conversations with the FDA, Percheron is on a mission to de-risk their drugs and attract strong partners. Listen in to understand how these innovative therapies could significantly impact the lives of patients with rare diseases, paving the way for a brighter future.