Ultragenyx Pharmaceutical (RARE) has received Breakthrough Therapy Designation from the FDA for GTX-102 as a treatment for Angelman syndrome. The decision is based on preliminary clinical evidence including positive data from the Phase 1/2 study in 74 patients with a full maternal UBE3A gene deletion.
Eric Crombez, chief medical officer at Ultragenyx, said: "Based on the strength of the Phase 2 data and with strong support and interest from the Angelman syndrome community, our Phase 3 Aspire study is rapidly enrolling across our global sites."
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May 22, 2026 14:46 ET Minutes of the latest Fed policy session was the highlight of the week along with survey data on the U.S. housing market. In Europe, survey data signaled the trends in the euro area private sector. Further, consumer price inflation data from the U.K. was in focus. In Asia, various economic indicators from China drew attention to the health of the economy.