In a groundbreaking first, a gene therapy in clinical trials has slowed the progression of Huntington's disease, a rare genetic disorder in which toxic bits of protein cause brain cells to malfunction and die.
To date, approved treatments for Huntington's disease aim to manage its symptoms, which most often emerge in a person's 30s or 40s. The progressive condition injures and kills key neurons involved in controlling mood, cognition and motor control. Various drugs can help to offset the depression, hallucinations and poorly coordinated movements that arise from that destruction.
