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Science and Pharmaceutical Updates

FAST works with leading researchers and pharmaceutical companies using cutting edge technology to accelerate our mission to find a cure for Angelman syndrome. At the Global Science Summit a few weeks ago, they shared an update on all of their work this past year that continues to confirm our mission to find a cure is possible.

Science Summit Update

https://www.youtube.com/watch?v=C2dDiEm7FFc&list=PLCTi8nkE1YPcaqcWDTrSB_NmVHXPJLo9G&index=1&t=18s

Keynote address: Cell & Gene Therapy Platforms

https://youtu.be/Cwt7BHUyWMcOverview of the Therapeutic Landscape for Angelman Syndromehttps://youtu.be/aA7cfmWr_KwCreation of a Robust Infrastructure Including Molecular, Neurobehavior & Biomarker Testinghttps://youtu.be/khVi7M059qoUpdate on AS Human IPSC Biorepository Project & the Angelman Syndrome Large Deletion Mouse Modelhttps://youtu.be/P2DqRGQTyl4

Engineering Human Cell Models for Multiple Angelman Syndrome (Epi)Genotypes

https://youtu.be/RSM0BepCWwMGene Therapy for Angelman Syndrome Through RNA Interferencehttps://youtu.be/Iw0lfS9pYNcTranslational Research in a Large Animal Model of Angelman Syndromehttps://youtu.be/H_kMcgmA-PY24/7 Emergency Care Hotlinehttps://youtu.be/OmPSqg53Q24Genetic Approaches for Treating Angelman Syndromehttps://youtu.be/2sq0PKQgfxQ

Hematopoietic Cell Gene Therapy for Angelman Syndrome

https://youtu.be/zlapDAdAwJAPanel Discussion with Researchers

Pharmaceutical Update

https://youtu.be/ulk7IKb9rtIKeynote address: Now Is the Time for Molecular Therapyhttps://youtu.be/_d9rpKHacN0Taysha Gene Therapies: Putting Patients at the Centerhttps://youtu.be/gfOgI3VOTGUNeuren Pharmaceuticals Ltd.: NNZ-2591 As A Potential Treatment For Angelman Syndromehttps://youtu.be/432S1RUevKoIonis Pharmaceuticals: Angelman Syndrome Program Updatehttps://youtu.be/qq6E9iMMicEGeneTx Biotherapeutics: GTX 102 Phase 1/2 Clinical Trial Updatehttps://youtu.be/65wZQf-WSI0Roche Pharmaceuticals: Angelman Syndrome Program Updatehttps://youtu.be/8_Qt0SVCHPwUltragenyx Pharmaceutical Inc.: Overcoming Challengeshttps://youtu.be/pm2KOr0xFacUpdate on the UK Natural History Studyhttps://youtu.be/diZkP1QC-W4

Panel Discussion with Pharma

Disclaimer

This website contains information for a broad audience and may include information on current and upcoming programs that are not yet approved or accessible The information provided is for general informational purposes only and is not intended as medical advice, diagnosis, or treatment. While FAST strives to provide accurate and up-to-date information, the content on this site may not always reflect the most current research or clinical guidelines. The inclusion of clinical trial information, treatments or specific healthcare providers does not imply endorsement, recommendation or guarantee of safety, efficacy, or availability. Reliance on any information provided by this website is solely at your own risk. FAST disclaims any liability for any errors or omissions in the information provided or for any decisions made based on this information. For personalized medical advice or specific health concerns including participation in any clinical trial, please consult a qualified healthcare professional.