Tag: FDA

A total of 101 posts are filed under FDA
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Kids (Six and Under) With Rare Blood Disorder, Chronic ITP, Get FDA Approval on New Novartis Drug, Promacta
Promacta® (eltrombopag) significantly increased and sustained platelet counts in two studies, including the largest Phase III clinical… Continue Reading
Today: Last Chance to Register for 21st Century Cures Initiative Briefing
Briefing on the 21st Century Cures Initiative: Second Discussion Draft takes place today, May 12, 2015 from 12:00PM – 1:30 PM. PhRMA… Continue Reading
Horizon Pharma’s Friedreich’s Ataxia Drug Gets Fast-Track Designation
Horizon Pharma PLC said Friday that its experimental Friedreich’s ataxia treatment has received fast-track designation from the U.S. Food… Continue Reading
Rare Disease Report: FDA Approves Unituxin (dinutuximab) for Neuroblastoma in Children
The U.S. Food and Drug Administration (FDA) has approved Unituxin (dinutuximab) as part of first-line therapy for pediatric patients with… Continue Reading
RAREcast: How the Orphan Drug Act Reshaped the Rare Disease Landscape
The U.S. Food and Drug Administration approved more than 230 new drugs to treat rare diseases in the past decade and there are currently… Continue Reading
Greater Patient Access to Fed-Funded Scientific Research? Coming in 2015
Rare disease patients, advocacy organizations, and researchers will soon have greater access to results from federally-funded scientific… Continue Reading
North Dakota Lawmakers Try to Help Family Fight Rare Disease
North Dakota lawmakers want to help a Grace city family who is struggling with a rare genetic disease. The Kulsrud brothers suffer from… Continue Reading
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