International Consortium Advances Angelman Syndrome Therapy
An international, multidisciplinary consortium is driving research into a microRNA-based therapy for Angelman Syndrome. Key institutions involved include the Angelman Syndrome Biomarker and Outcome Measure Consortium (AS-BOMC), Boston Children’s Hospital, and leading academic and medical centers, private research partners, and international organizations.
The University of North Carolina at Chapel Hill, Duke University, University of California, San Diego, and the University of Pennsylvania are among the prominent academic institutions contributing to the research. Private partners such as Ionis Pharmaceuticals, Rarebase, and Ultragenyx Pharmaceutical are also playing significant roles. The Angelman Syndrome Alliance in the UK and Europe, along with universities and research centers in Europe and Asia, are involved in international collaborations.
The Foundation for Angelman Syndrome Therapeutics (FAST) is actively supporting this research, funding and coordinating numerous projects. A recent collaboration aims to expedite data analysis and publication from ongoing and previous natural history studies, involving RTI International and Boston Children’s Hospital. The goal is to better understand Angelman Syndrome’s common features over time, improve clinical trial outcomes, and enhance patient care. The current study, funded by the U.S. Food and Drug Administration, is recruiting up to 150 patients at seven U.S. sites and is expected to conclude in August 2022.
The international research partnership for Angelman Syndrome microRNA therapy involves a wide range of institutions, from academic and medical centers to private research partners and international organizations. The ongoing study, funded by the U.S. Food and Drug Administration, aims to improve clinical trial outcomes and patient care. The collaboration between FAST, RTI International, and Boston Children’s Hospital seeks to expedite data analysis and publication, further advancing the understanding and treatment of Angelman Syndrome.
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