What Is Angelman Syndrome? Everything to Know About the Rare Genetic Disorder Colin Farrell’s Son James Has
For the first time, Colin Farrell has opened up his home and talked in-depth about life with his son James, who has Angelman ...
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 ...
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 ...
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 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 ...
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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 ...
Business Insider2d
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, ...
Stoneham Independent15d
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 ...
GoodNewsNetwork4d
4-Year-old Girl Told She’d ‘Never Walk’ takes Her First Steps–With Sisters Screaming as They Shoot Video
A four-year-old girl with DUP15q syndrome, who doctors said would never walk, has defied expectations to take her first steps.
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 ...
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 ...