Always Fatal Huntington’s Disease Is Successfully Treated For First Time With Gene Therapy

Always Fatal Huntington’s Disease Is Successfully Treated For First Time With Gene Therapy

Always Fatal Huntington’s Disease Is Successfully Treated For First Time With Gene Therapy Co-founders of the UCL Huntington’s Disease Centre – Professors Tabrizi and Wild There is no cure for Huntington’s disease, a devastating brain disorder that causes severe motor loss, dementia, and eventual death. But, a clinical trial delivered positive results this week, finding that patients receiving a new treatment for 36 months experienced 75% less progression of the disease overall (compared to patients who did not receive the treatment). This is the first time a drug trial achieved a significant slowing of Huntington’s progression, according to uniQurea, a gene therapy company based in the Netherlands and US. The new gene therapy, AMT-130, was performed on 29 patients including 12 who were given a high dose. Those given a high dosage of AMT-130 experienced 75% less disease progression, as measured by a standard rating scale that incorporates motor, cognitive, and functional measures. There was…