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Newsweek News source @newsweek.com · New York 🇺🇸· Mar 19 ·

#RareDiseases #MPS #FDAApproval

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Moms stage "funeral" outside FDA in drug protest
More than 100 mothers and advocates affected by a type of rare disease gathered outside the Food and Drug Administration (FDA) on Wednesday to stage a funeral to "mourn the futures" of their children, following the agency's rejections of various rare disease treatments.
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U.S. Food and Drug Administration News source @fda.gov · Silver Spring, Maryland 🇺🇸· Jan 5 ·

#PediatricHealth #RareDiseases #DrugApproval

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FDA approves therapy for rare blood disorder in pediatric patients 12
This action marks first drug approved for pediatric patients with acquired thrombotic thrombocytopenic purpura The U.