Scientific and Clinical Research
for Angelman syndrome
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Research is advancing rapidly to better understand and develop a treatment for Angelman Syndrome.
Research is advancing rapidly to better understand and develop a treatment for Angelman Syndrome.
The Research Pipeline for Angelman Syndrome
The Research Pipeline for Angelman Syndrome
Currently, there are 24 research programs. The largest funders are global patient organizations such as FAST, ASF, and ASA. Four of these programs are already in the clinical research phase (study of experimental drugs in humans):
2 that use a disease modification strategy via the ASO technique, and
2 that target the effects resulting from the non-functional gene.
Updates on the progress of preclinical and clinical research are provided annually at the FAST North American Scientific Foundation's Congress. The presentations are recorded and made available on YouTube.
Updates on the progress of preclinical and clinical research are provided annually at the FAST North American Scientific Foundation's Congress. The presentations are recorded and made available on YouTube.
How to activate Portuguese subtitles on YouTube:Click play on the video. In the lower bar of YouTube, click on the "subtitles/closed captions (c)" icon to activate.Click on the "details" icon next to it. Select "automatically translate" > Portuguese.
Educational Videos by FAST (Foundation for Angelman Syndrome Therapeutics - USA)
Educational Videos by FAST (Foundation for Angelman Syndrome Therapeutics - USA)
2022 What Parents Need to Know About Trial Participation
2022 What Parents Need to Know About Trial Participation
Jennifer Panagoulias
Co-diretora A-BOM
FAST Science Summit (USA)
2023 Patient-Focused Pharmaceutical Development
2023 Patient-Focused Pharmaceutical Development
Ryan Fischer
COO of FAST
FAST Science Summit (USA)
2023 Full Playlist
2023 Full Playlist
Videos from the FAST Global Scientific Conference and Gala
FAST Science Summit (USA)
2022 Full Playlist
2022 Full Playlist
Videos from the FAST Global Scientific Conference and Gala
FAST Science Summit (USA)
ULTRAGENYX: updates on the clinical study of the experimental drug GTX-102
ULTRAGENYX: updates on the clinical study of the experimental drug GTX-102
For the most up-to-date information on this clinical study, visit: https://clinicaltrials.gov/study/NCT04259281
Latest videos are listed first:
2023 GTX-102 UPDATE
2023 GTX-102 UPDATE
Kemi Olugemo
VP of Ultragenyx Pharmaceuticals
FAST Science Summit (USA)
2022 GTX-102 UPDATE
2022 GTX-102 UPDATE
Kemi Olugemo
VP of Ultragenyx Pharmaceuticals
FAST Science Summit (USA)
2021 GTX-102 UPDATE
2021 GTX-102 UPDATE
Emil Kakkis
CEO of Ultragenyx Pharmaceuticals
FAST Science Summit (USA)
IONIS: updates on the HALOS clinical study, experimental drug ION582
IONIS: updates on the HALOS clinical study, experimental drug ION582
For the most up-to-date information on this clinical study, visit: https://clinicaltrials.gov/study/NCT05127226
Latest videos are listed first:
2023 HALOS UPDATE
2023 HALOS UPDATE
Rebecca Crean
Executive Director of Clinical Development at Ionis
FAST Science Summit (USA)
2022 HALOS UPDATE
2022 HALOS UPDATE
Rebecca Crean
Executive Director of Clinical Development at Ionis
FAST Science Summit (USA)
2021 HALOS UPDATE
2021 HALOS UPDATE
Frank Bennett
Senior VP of Ionis Pharmaceuticals
FAST Science Summit (USA)
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©2024 GAMAS
©2024 GAMAS
All Rights Reserved.Original content and images cannot be shared without previous official permission from GAMAS. Images: Adobe Firefly, Freepik.
All Rights Reserved.Original content and images cannot be shared without previous official permission from GAMAS. Images: Adobe Firefly, Freepik.
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