Fred Cheng celebrates birthday by learning more about Angelman Syndrome
Fred Cheng recently celebrated turning 41 by revealing that his focus this year has been on son Asher. On 11 November, the ...
Ultragenyx posts trial data backing Phase 3 design for Angelman syndrome therapy
Ultragenyx Pharmaceutical (RARE) shows promising results for GTX-102 in Angelman syndrome Phase 1/2 trial, supporting a Phase ...
Ultragenyx Presents Positive Update on GTX-102 Angelman Syndrome Program at FAST’s 17th Annual Global Science Summit
Phase 1/2 data show improvements across all domains and confirm that Phase 3 Aspire study is amply powered to establish ...
Ultragenyx Presents Positive Update On GTX-102 Angelman Syndrome Program
Ultragenyx Pharmaceutical Inc. (RARE) announced Phase 1/2 data in support of the Phase 3 Aspire study for GTX-102, its ...
Morningstar4d
Ultragenyx Presents Positive Update on GTX-102 Angelman Syndrome Program at FAST’s 17th Annual Global Science Summit
The global Phase 3 Aspire study will enroll approximately 120 patients with Angelman syndrome with a genetically confirmed diagnosis of full maternal UBE3A gene deletion and will include a 48-week ...
Morningstar7d
Encoded Demonstrates Therapeutic Potential of a Vectorized miRNA-based Approach for Angelman Syndrome at the FAST Annual Global Science Summit
New preclinical data for candidate ETX201 illustrate its potential as a first-in-class gene therapy for Angelman syndrome. Studies in non-human primates demonstrate safety, biodistribution ...
GlobalData on MSN6d
Ionis announces design for Phase III trial of Angelman syndrome therapy
Ionis has announced the design for a Phase III trial of ION582, a treatment for Angelman syndrome (AS), following discussions with the FDA.
Ionis Announces Pivotal Phase 3 Trial Design for ION582 in Angelman Syndrome
Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced the pivotal Phase 3 study design following successful alignment with the U.S. Food and Drug Administration (FDA) on ION582, an ...