A major step forward for AHC research. Researchers at Brown University have developed and characterized a genetically engineered C. elegans model for ATP1A3-related disease that enables scalable drug screening across multiple patient variants. The work, led by Dr. Anne Hart and Diana Wall, represents an important advance toward accelerating therapeutic discovery in AHC. These models are valuable not only for advancing the mechanistic science of AHC, but also for helping bridge basic disease biology with therapeutic screening and drug discovery.
RARE Hope is grateful to @cureahc @ahckids, Hope4Livi and @forhenryahc for their partnership on this project. 
