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 ...
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 ...
Nasdaq

GeneTx and Ultragenyx Announce FDA has Removed Clinical Hold on Phase 1/2 Clinical Study of GTX-102 for the Treatment of Angelman Syndrome in the U.S.

SARASOTA, Fla. and NOVATO, Calif., Sept. 27, 2021 (GLOBE NEWSWIRE) -- GeneTx Biotherapeutics LLC and Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), companies partnered in the development of GTX-102, ...