Science And Pharmaceutical Updates - FAST

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

Keynote address: Cell & Gene Therapy Platforms

Overview of the Therapeutic Landscape for Angelman Syndrome

Creation of a Robust Infrastructure Including Molecular, Neurobehavior & Biomarker Testing

Update on AS Human IPSC Biorepository Project & the Angelman Syndrome Large Deletion Mouse Model

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

Gene Therapy for Angelman Syndrome Through RNA Interference

Translational Research in a Large Animal Model of Angelman Syndrome

24/7 Emergency Care Hotline

Genetic Approaches for Treating Angelman Syndrome

Hematopoietic Cell Gene Therapy for Angelman Syndrome

Panel Discussion with Researchers

Pharmaceutical Update

Keynote address: Now Is the Time for Molecular Therapy

Taysha Gene Therapies: Putting Patients at the Center

Neuren Pharmaceuticals Ltd.: NNZ-2591 As A Potential Treatment For Angelman Syndrome

Ionis Pharmaceuticals: Angelman Syndrome Program Update

GeneTx Biotherapeutics: GTX 102 Phase 1/2 Clinical Trial Update

Roche Pharmaceuticals: Angelman Syndrome Program Update

Ultragenyx Pharmaceutical Inc.: Overcoming Challenges

Update on the UK Natural History Study

Panel Discussion with Pharma