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Angelman Syndrome News We are dedicated to sharing the latest news, research, and Angelman syndrome patient perspectives.

Angelman Syndrome News is a digital platform providing daily updates on research, science and advice news for Angelman Syndrome patients and caregivers.

The U.S. FDA has granted breakthrough therapy designation to GTX-102, Ultragenyx’s investigational gene therapy for Ange...
02/07/2025

The U.S. FDA has granted breakthrough therapy designation to GTX-102, Ultragenyx’s investigational gene therapy for Angelman syndrome. https://buff.ly/fJ0gZmz

Columnist Joelene Wand has learned some strategic and efficient thinking can let her continue supporting friends and fam...
10/06/2025

Columnist Joelene Wand has learned some strategic and efficient thinking can let her continue supporting friends and family while caregiving. https://buff.ly/6pQUaDk

The FDA has cleared Mavrix Bio to start a first-in-human trial of MVX-220, the company's experimental gene therapy for A...
04/06/2025

The FDA has cleared Mavrix Bio to start a first-in-human trial of MVX-220, the company's experimental gene therapy for Angelman syndrome. https://buff.ly/zyReqmc

Treatment with octanoic acid, a fatty acid in breast milk, reactivated a gene that's normally suppressed, leading to gai...
07/05/2025

Treatment with octanoic acid, a fatty acid in breast milk, reactivated a gene that's normally suppressed, leading to gains in cognition. https://buff.ly/abaywzM

One year after her son's Angelman syndrome diagnosis, columnist Joelene Wand shares the pieces of wisdom that have helpe...
08/04/2025

One year after her son's Angelman syndrome diagnosis, columnist Joelene Wand shares the pieces of wisdom that have helped her cope. https://buff.ly/QKFNI4L

Those with the most common Angelman-causing gene defect had more autistic traits than those with other disease-causing a...
02/04/2025

Those with the most common Angelman-causing gene defect had more autistic traits than those with other disease-causing abnormalities. https://buff.ly/2BAR3TP

Columnist Joelene Wand says she's constantly dealing with caregiver guilt while raising a son with Angelman. But she's l...
11/03/2025

Columnist Joelene Wand says she's constantly dealing with caregiver guilt while raising a son with Angelman. But she's learning acceptance. https://buff.ly/15GMvML

A report details three people with Angelman syndrome and gelastic syncope episodes, in which they laugh to the point of ...
05/03/2025

A report details three people with Angelman syndrome and gelastic syncope episodes, in which they laugh to the point of blanking out. https://buff.ly/7cPr0v7

Wearing blue and raising awareness are great ways to honor International Angelman Day on Feb. 15, writes columnist Joele...
11/02/2025

Wearing blue and raising awareness are great ways to honor International Angelman Day on Feb. 15, writes columnist Joelene Wand. https://buff.ly/4hSszuz

Supporters and organizations prepare to raise awareness of Angelman syndrome on Feb. 15, International Angelman Day. htt...
05/02/2025

Supporters and organizations prepare to raise awareness of Angelman syndrome on Feb. 15, International Angelman Day. https://buff.ly/4aLQ7PA

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