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

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Keynote address: Stem Cell & Gene Therapy Platforms

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Overview of the Therapeutic Landscape for Angelman Syndrome
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Creation of a Robust Infrastructure Including Molecular, Neurobehavior & Biomarker Testing
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Update on AS Human IPSC Biorepository Project & the Angelman Syndrome Large Deletion Mouse Model
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Engineering Human Stem Cell Models for Multiple Angelman Syndrome (Epi)Genotypes
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Gene Therapy for Angelman Syndrome Through RNA Interference
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Translational Research in a Large Animal Model of Angelman Syndrome
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24/7 Emergency Care Hotline
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Genetic Approaches for Treating Angelman Syndrome
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Hematopoietic Stem Cell Gene Therapy for Angelman Syndrome
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Panel Discussion with Researchers

Pharmaceutical Update

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Keynote address: Now Is the Time for Molecular Therapy
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Taysha Gene Therapies: Putting Patients at the Center
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Neuren Pharmaceuticals Ltd.: NNZ-2591 As A Potential Treatment For Angelman Syndrome
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Ionis Pharmaceuticals: Angelman Syndrome Program Update
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GeneTx Biotherapeutics: GTX 102 Phase 1/2 Clinical Trial Update
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Roche Pharmaceuticals: Angelman Syndrome Program Update
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Ultragenyx Pharmaceutical Inc.: Overcoming Challenges
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Update on the UK Natural History Study
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Panel Discussion with Pharma

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