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Ultragenyx Pharmaceutical Receives Breakthrough Therapy Designation from FDA for GTX-102 in Treating Angelman Syndrome
Ultragenyx Pharmaceutical Inc. has made a significant breakthrough in the treatment of Angelman syndrome after receiving Breakthrough Therapy Designation from the U.S. Food and Drug Administration (FDA) for its drug GTX-102 (apazunersen). This designation is a major milestone for the company, as it aims to expedite the development and