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Precision Medicine for Rare Diseases: Julia Vitarello’s Fight for Change_e.061
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This episode of Lab Rats to Unicorns features an inspiring conversation with Julia Vitarello, founder of Mila’s Miracle Foundation and a trailblazer in the field of individualized genetic therapies. Julia shares the story of her daughter Mila, who was diagnosed with a rare and fatal genetic disease, Batten disease, at age six. Confronted with a system ill-equipped to handle ultra-rare conditions, Julia embarked on a transformative journey to develop Milasen, the first-ever genetic therapy tailored for a single individual.
Through resilience and collaboration, Julia mobilized a global network of scientists, regulators, and supporters to create a treatment that set a precedent for a new approach in medicine. Beyond her daughter’s story, Julia reflects on the systemic barriers families face when accessing life-saving treatments and how her foundation is addressing these challenges. She explores the future of healthcare, advocating for changes to regulatory and reimbursement models to scale access to innovative therapies for rare disease communities. Julia’s pioneering work is a testament to the power of advocacy, science, and the unwavering determination of a mother to spark a revolution in medicine.
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