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N-of-1 therapies: How clinical trials for a single patient can change the future

Q&A with advocate Julia Vitarello on an emerging pathway for developing therapies for ultrarare diseases

At age six, Mila Makovec was diagnosed with Batten disease, a rare and progressive neurological disorder. Upon learning that her daughter’s disease had no cure, Julia Vitarello started Mila’s Miracle Foundation to initiate and fund novel treatments. Working alongside Dr. Timothy Yu and his team at Boston Children’s Hospital to develop a truly personalized treatment for Mila, Julia found herself on the front lines of ultrarare drug development, helping to pioneer a movement for “n-of-1″ therapies – treatments designed for one person’s unique genetic mutation.

In a remarkable collaboration between scientists, physicians, drug developers, foundations and regulators, a novel medicine was created, tested and delivered in just one year from Mila’s diagnosis. Mila became the first person in the world to receive a drug customized for just one person. It was named milasen.

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