A gene therapy targeting organs beyond the brain could improve quality of life and possibly extend survival in children with the neurodegenerative disease.
Vertex Pharmaceuticals’ drug, Journavx, blocks a sodium channel outside of the brain. Experts hope that this non-addictive pain drug may be the first of many.
The protein APLP1 strongly associates with brain-derived EVs. A blood test could one day isolate these molecular packets to diagnose neurological disorders.