Ultragenyx Pharmaceutical (RARE) shows promising results for GTX-102 in Angelman syndrome Phase 1/2 trial, supporting a Phase ...

Nov. 09, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced Phase 1/2 data in support of ...

"Ionis announces design for Phase III trial of Angelman syndrome therapy" was originally created and published by Clinical Trials Arena, a GlobalData owned brand.

Overall, 97% of people in the medium and high dose groups assessed in the study saw an improvement in overall AS symptoms as measured by the Angelman Syndrome Clinical Global Improvement Change ...

A four-year-old girl who doctors said would never walk has defied the odds and taken her first steps. Scottie Mae Blair was ...

A four-year-old girl who doctors said would never walk has defied the odds and taken her first steps. Scottie Mae Blair was ...

CARLSBAD, Calif. - Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) has confirmed plans to initiate a Phase 3 study for ION582, a potential treatment for Angelman syndrome (AS), in the first half of 2025.

Scottie Mae Blair was diagnosed with Angelman syndrome at 11-months-old. The rare genetic condition causes severe physical and learning disabilities. Upon the diagnosis, parents Logan Blair, 32, and ...

Third quarter 2024 total revenue grew 42% versus prior year to $139 million, including Crysvita® revenue of $98 million and Dojolvi® revenue of ...

Off a rural road in Medina County is a little church in Valley City where a women’s ministry is having a big impact far beyond the four walls of their church.

Ionis Pharmaceuticals (IONS) announced the pivotal Phase 3 study design following successful alignment with the U.S. FDA on ION582, an ...