The Way Forward for Mechanism‐Based Therapeutics in Genetically Defined Neurodevelopmental Disorders
Abstract
Rare genetically defined neurodevelopmental disorders with increased risk of autism have recently become an entry point for autism‐related drug discovery. Through exploration of downstream effects of the pathological mutations, specific mechanistic pathways have been identified as dysregulated. The identification of shared mechanisms across forms of autism opens the door for the development of novel “mechanism‐based therapeutics.” However, confidence in the therapeutic mechanism does not diminish the need for well‐designed clinical trials.
Document Details
- Document Type
- Pub Defense Publication
- Publication Date
- Aug 12, 2018
- Source ID
- 10.1002/cpt.1181
Entities
People
- Meera E. Modi
- Mustafa Şahin
Organizations
- Autism Speaks
- Harvard Medical School
- Hoffmann-La Roche
- Nancy Lurie Marks Family Foundation
- National Institutes of Health
- Novartis
- Pfizer
- Tuberous Sclerosis Alliance
- United States Department of Defense