Medicine: Adopting Orphan Drugs

Congress tries to encourage new remedies for rare diseases

In the summer of 1974, Concert Pianist Sharon Roubeck Dobkin suffered a series of seizures that left her with uncontrollable tremors, an inability to use her arms and legs or even hold her head upright. Two years and many doctors later, Dr. Melvin Van Woert of New York City's Mount Sinai Hospital identified the condition that ended her career: myoclonus, a nervous disorder that affects only 2,000 Americans. Van Woert had received a series of grants and special permission from the FDA to treat the...

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