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Human IPSC-Based Drug Testing Platform for Duchenne Muscular Dystrophy

Unmet NeedThe development of new drugs is a capital and time intensive process. For this reason, the development of orphan drugs for those diseases that affect a small subset of the population. One of such diseases, Duchenne’s muscular dystrophy (DMD) affects 1 in 5000 boys worldwide and has no known cure. Current drug screen platforms include animal...

Published: 3/14/2025 | Inventor(s): Gabsang Lee, Kathryn Wagner, Congshan (Sunny) Sun

Keywords(s): Discovery/Research Tools, Disease Indication, In Vitro Research Tool, Muscular Disorders, Small Molecules, Therapeutic Matter, Therapeutic Substance, Therapeutics

Category(s): Clinical and Disease Specializations > Rare Diseases, Technology Classifications > Therapeutic Modalities > Small Molecules, Technology Classifications > Research Tools > Research Stem Cells