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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.

Dosing has begun in a Phase 3 clinical trial testing GTX-102 (apazunersen) in a broader population of people with Angelm...
03/11/2025

Dosing has begun in a Phase 3 clinical trial testing GTX-102 (apazunersen) in a broader population of people with Angelman syndrome. https://buff.ly/6K2MAH8

For columnist Joelene Wand, one of the biggest challenges of raising a nonverbal child is the uncertainty about how they...
07/10/2025

For columnist Joelene Wand, one of the biggest challenges of raising a nonverbal child is the uncertainty about how they're really feeling. https://buff.ly/QSDyCww

The investigational gene therapy MVX-220 has earned fast track designation in the U.S. for the treatment of Angelman syn...
01/10/2025

The investigational gene therapy MVX-220 has earned fast track designation in the U.S. for the treatment of Angelman syndrome. https://buff.ly/zVXgvbo

GTX-102 is an antisense oligonucleotide with genetic material that's designed to inhibit a mechanism that silences the p...
06/08/2025

GTX-102 is an antisense oligonucleotide with genetic material that's designed to inhibit a mechanism that silences the paternal UBE3A gene. https://buff.ly/dK6cItK

People sometimes say insensitive things about columnist Joelene Wand's nonverbal son. She must advocate when he's being ...
22/07/2025

People sometimes say insensitive things about columnist Joelene Wand's nonverbal son. She must advocate when he's being "othered." https://buff.ly/CSi6daJ

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

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