Ultragenyx posts trial data backing Phase 3 design for Angelman syndrome therapy
Ultragenyx Pharmaceutical (NASDAQ:RARE) announced Saturday that GTX-102, its candidate for the rare neuro-genetic disorder ...
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 ...
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 ...
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Colin Farrell explains how son with Angelman syndrome helped ‘fuel’ his sobriety journey
Colin Farrell’s son James had a part to play in his sobriety journey. The now-21-year-old has Angelman syndrome, and the actor called his child’s diagnosis “fuel” in choosing to abstain fr ...
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 ...
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Tot told she would never walk takes her first steps
A four-year-old girl who doctors said would never walk has defied the odds and taken her first steps. Scottie Mae Blair was ...
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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 ...
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Ultragenyx’s GTX-102 Program Gains Momentum: Buy Rating Affirmed Amidst Promising Trial Results and Sales Projections
Joseph Schwartz has given his Buy rating due to a combination of factors surrounding Ultragenyx Pharmaceutical’s GTX-102 program for Angelman syndrome. The updated data from the Phase 1/2 trial, ...
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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 ...
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Ultragenyx Presents Positive Update On GTX-102 Angelman Syndrome Program
(RTTNews) - Ultragenyx Pharmaceutical Inc. (RARE) announced Phase 1/2 data in support of the Phase 3 Aspire study for GTX-102, its investigational antisense oligonucleotide for Angelman syndrome.