Tag: FDA

A total of 135 posts are filed under FDA
CheckRare: FDA Approves Haegarda to Treat HAE
The U.S. Food and Drug Administration today approved Haegarda, the first C1 Esterase Inhibitor (Human) for subcutaneous administration to… Continue Reading
Rare Disease Report: RASRx is the Latest Duchenne Drug To Get Orphan Status From the FDA
Thanks to Rare Disease Report for this piece. University of Southern California (USC) start-up RASRx hopes to develop a drug to treat… Continue Reading
Gottlieb’s Aggressive Plans to Streamline the Orphan Drug Review Process
Thanks to Rare Disease Report for this article. On June 20, 2017, Food and Drug Administration (FDA) Commissioner Scott Gottlieb,… Continue Reading
Rare Disease Patient and Lawyer: “I Want to Walk Again”
By Paula J. Owen (Source) It is a rare, inherited, progressive disease affecting one in 50,000 that results in loss of ambulation six to… Continue Reading
ProQR Receives Fast Track Designation from the FDA for QR-110 for Leber’s Congenital Amaurosis Type 10
 ProQR Therapeutics N.V.(Nasdaq:PRQR) today announced that it received Fast Track designation from the U.S. Food and Drug… Continue Reading
FDA Grants Priority Review Status to Ultragenyx
Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for rare and… Continue Reading
NYT: Senate Confirms Scott Gottlieb to Head F.D.A.
The Senate voted 57 to 42 on Tuesday to confirm Dr. Scott Gottlieb as commissioner of the Food and Drug Administration, where he will be… Continue Reading
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