FDA Press Release Link: FDA Rare Disease Innovation Hub to Enhance and Advance Outcomes for Patients | FDA By: Patrizia Cavazzoni, MD, Director, Center for Drug Evaluation and Research and Peter Marks, MD, PhD, Director, Center for Biologics Evaluation and Research An estimated 10,000+ rare diseases affect more than 30 million people – approximately one …
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FDA
NPC Parents Help Get First Drug Recommendation!
A Food and Drug Administration (FDA) advisory panel recommended Zavesca to treat NPC. Phil Marella of DART, Cindy Parseghian of the Parseghian Foundation and Barb Vorpahl of the NNPDF spoke during the public portion of the hearing. The panel of outside medical experts voted 10-to-3 in favor of a question that asked “in light of …