Rare Disease Through a Different Lens

In 2017, the U.S. Food and Drug Administration’s Center for Drug Evaluation and Research (CDER) approved many new, innovative therapies for patients with rare diseases. These approvals included the first treatment for Batten disease, a rare and fatal disorder of the nervous system. Here, CDER Director Janet Woodcock, M.D., gives her perspective, along with a Batten disease advocate, an FDA statistician, and an FDA pediatrician.

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