Gene Therapy + PDE with Dr. Phillip Pearl

Pediatric neurologist Dr. Phillip Pearl joins Cure PDE to explore the rapidly evolving field of gene therapy and what advances in other rare neurological disorders may teach us about the future of PDE treatment.

Drawing on his experience with gene therapy for Aromatic L-Amino Acid Decarboxylase (AADC) deficiency, Dr. Pearl shares insights into how similar therapeutic approaches could one day inform research and treatment development for pyridoxine-dependent epilepsy.

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Cure PDE Family Retreats

Our Family Retreats bring families from across the United States and Canada together for connection, education, research updates, and time with others who understand the PDE journey.

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Cure PDE brings families, researchers, clinicians, and advocates together throughout the year through in-person gatherings and educational conversations.

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