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GeneTx and Ultragenyx Provide Preliminary Update on Phase 1/2 Clinical Study of GTX-102 in Canada and U.K. Patients with Angelman Syndrome
SARASOTA, Fla. and NOVATO, Calif., Jan. 05, 2022 (GLOBE NEWSWIRE) -- GeneTx Biotherapeutics LLC and Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), companies
About this update from Ultragenyx Pharmaceutical Inc.
[{"type":"text","content":"SARASOTA, Fla. and NOVATO, Calif., Jan. 05, 2022 (GLOBE NEWSWIRE) -- GeneTx Biotherapeutics LLC and Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), companies partnered in the development of GTX-102, an investigational treatment for Angelman syndrome, today announced that the first four patients in the Phase 1/2 study have received multiple doses of GTX-102 and regular assessments for safety. To date three have also received a preliminary assessment of clinical response. There have been no treatment-related serious adverse events of any type nor adverse events related to lower extremity weakness observed in these patients, and initial assessments have shown early signs of clinical activity. The data safety monitoring board (DSMB) met to discuss the assessments for the first two patients in Cohort 4 (ages 4 to","length":1045,"tagName":"div"}]