Nature

Genetic mechanisms leading to Angelman syndrome

The rectangles represent chromosome 15. Hatched chromosomes have a paternal pattern of gene functioning and DNA methylation; open chromosomes have a maternal pattern. Angelman syndrome is caused by a ...
CNN

GeneTx and Ultragenyx Announce Investigational New Drug (IND) Application Active for GTX-102 in Patients with Angelman Syndrome

SARASOTA, Fla. and NOVATO, Calif., Jan. 15, 2020 (GLOBE NEWSWIRE) -- GeneTx Biotherapeutics LLC and Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the ...
Seeking Alpha

Ultragenyx Announces Completion of Enrollment in Global Phase 1/2 Trial of GTX-102 in Pediatric Patients with Angelman Syndrome

NOVATO, Calif., Jan. 03, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious ...
Seeking Alpha

Ultragenyx Receives Breakthrough Therapy Designation for GTX-102 in Angelman Syndrome

NOVATO, Calif., June 27, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (RARE), today announced that it has received Breakthrough Therapy Designation from the U.S. Food and Drug ...
Nasdaq

GeneTx and Ultragenyx Receive Clearance from Health Canada to Begin Clinical Study of GTX-102 in Canada for the Treatment of Angelman Syndrome

SARASOTA, Fla. and NOVATO, Calif., May 19, 2021 (GLOBE NEWSWIRE) -- GeneTx Biotherapeutics LLC and Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), companies partnered in the development of ...