Addressing Challenges in Rare Disease Drug Development - Together

Dr. Richard Moscicki, Deputy Center Director for Science Operations at the Center for Drug Evaluation and Research at the Food and Drug Administration (FDA), noted in his remarks at the FDA public...
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Rare Disease Community Loses a Champion

The rare disease community lost a champion last week when Sam Berns passed away at age 17 due to complications from progeria, an extremely rare genetic disease which causes accelerated aging. Last...
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