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GeneTx and Ultragenyx Announce Positive Interim Phase 1/2 Data

GeneTx and Ultragenyx Announce Positive Interim Phase 1/2 Data

“So far we are seeing rapid improvements in multiple areas, including some kids doing things they’ve never done before, and I don’t believe this rate of progress in development skills has been seen before in Angelman syndrome,” commented Elizabeth M. Berry-Kravis, M.D. investigator in the GTX-102 clinical study.

Angelman syndrome biobank

Angelman syndrome biobank

The first biobank in Australia aiming to improve research and treatments into rare genetic diseases caused by changes to genes on chromosome 15, including Prader-Willi Syndrome and Angelman Syndrome, will be established at the Murdoch Children’s Research Institute (MCRI). 

Roche Clinical trial

Roche Clinical trial

Roche and Genentech (the US member of the Roche group) have recruited the first patient in their Phase 1 clinical trial named "Tangelo" that is targeting AS with an oligonucleotide.

News & Media

News & Media

Australian National Rugby League (NRL) identities Damien Cook, Iosia Soliola, Sam Perrett and Curtis Rona have been using their public profiles to increase awareness into Angelman syndrome.

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Enabling Research

Enabling Research

The Global Angelman Syndrome Registry is creating new opportunities, insight and understanding into Angelman syndrome, providing an invaluable tool for facilitating research.

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Raising Awareness

Raising Awareness

This beautiful video is one of a series produced to illustrate common features of Angelman syndrome and highlights individuality amongst those who have been diagnosed.

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About the Foundation

The Foundation for Angelman Syndrome Therapeutics Australia (or FAST Australia) is an organisation of families and professionals dedicated to assisting individuals living with Angelman syndrome to realise their full potential and quality of life through funding research, education and advocacy. Our focus is treatments that will improve the symptoms of Angelman syndrome and ultimately research that will provide a cure.

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Newly diagnosed

Resources to help those new to Angelman syndrome.

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Support projects progressing Angelman syndrome research.

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