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There is no effective treatment for the rare genetic disorder fragile X syndrome, so two parents created a foundation to fund research. But they found there's no easy road to a cure.
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Tired of waiting for a cure for breast cancer, a coalition of activists now leans hard on Congress to steer money to particular research projects. Critics say that approach may miss promising leads.
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An international team of scientists is experimenting with a potential drug to block the production of a protein linked to amyotrophic lateral sclerosis.
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New drugs are usually tested in animals before they're tested in humans. But many of those studies aren't done carefully enough, analysts say. So time and money is wasted, and treatments are delayed.