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Multi-Method, Multi-Stakeholder Approaches to Advancing Research and Clinical Trial Readiness in Rare Neurogenetic Conditions: A Focus on ASXL-Related Disorders

https://youtu.be/PCRVv2uZ_ok The identification of rare monogenic causes of neurodevelopmental disabilities has surged with advances in next-generation sequencing and broader access to genetic testing. This progress has paved the way for the development of novel disease-modifying therapies that address the root…

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