An investigational gene therapy for a rare neurodegenerative disease that begins in early childhood, known as giant axonal neuropathy (GAN), was well tolerated and showed signs of therapeutic benefit in a clinical trial led by the National Institutes of Health.
Prion Disease Rising in the U.S.
The incidence of Creutzfeldt-Jakob disease (CJD), a universally fatal prion disease that progresses rapidly, rose consistently from 2007 to 2020, a study of death certificates
December 12, 2023