GeneTx and Ultragenyx Announce Orphan Drug Designation and Rare Pediatric Disease Designation for GTX-102
Ultragenyx Pharmaceutical Inc. (RARE)
Last ultragenyx pharmaceutical inc. earnings: 2/13 04:01 pm
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Source: GlobeNewswire
SARASOTA, Fla. and NOVATO, Calif., Sept. 03, 2019 (GLOBE NEWSWIRE) -- GeneTx Biotherapeutics LLC and Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation and Rare Pediatric Disease Designation to GTX-102 for the treatment of Angelman Syndrome, a serious, debilitating rare neurogenetic disorder that affects approximately 1 in 15,000 people worldwide. “The granting of these designations from the FDA is an enormous milestone for the Angelman community,” stated Paula Evans, Chief Executive Officer of GeneTx. “We are thrilled that the FDA recognizes the urgency for the development of effective therapeutics in this patient population and that GeneTx,
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