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GeneTx and Ultragenyx Announce Positive Interim Phase 1/2 Data on Investigational GTX-102 Demonstrating Improvement in Patients with Angelman Syndrome

SARASOTA, Fla. and NOVATO, Calif., Oct. 26, 2020 (GLOBE NEWSWIRE) — GeneTx Biotherapeutics LLC and Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), companies partnered in the development of intrathecally administered GTX-102, an investigational treatment for Angelman syndrome, today announced positive interim data from the Phase 1/2 study of GTX-102. Preliminary results from the first five patients treated […]

Recent Posts

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Scientists uncover secrets of brain development: Extensive neural migration continues after birth

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