Tag Archives: rarediseses

Four American families are desperate to save their children from a devastating genetic disease. The cure exists — but it’s up to them to raise $1.15 million to fund a clinical trial. Each of these families has a child with spastic paraplegia 50 (SPG50), a rare neurological disorder that affects …

Scientists may have discovered the first therapy for Huntington’s disease, a brain disorder that until now has had no effective treatments. Researchers at the University of College London (UCL) announced positive results from a global clinical trial for a new gene therapy, AMT-130. Developed by the Dutch biotechnology company uniQure, …