Lack of continued help for poor families involved in Huntington’s-disease research has sown resentment and mistrust, says Ignacio Muñoz-Sanjuan.
By Ignacio Muñoz-Sanjuan
After decades of research, a genetic therapy for Huntington’s disease is being tested in clinical trials. Sponsored by Swiss pharmaceutical firm Roche and US-based Ionis Pharmaceuticals, this trial targets the gene that causes the disease. If the new treatment works, it could offer a way to halt progression of this genetic disease — an awful neurodegenerative disorder that attacks mainly the brain. Huntington’s is caused by a single gene mutation transmitted in a dominant fashion, so a child has a 50% chance of inheriting the condition if one of their parents carries a single copy of the defective gene. KEEP READING.
FUENTE: Nature Magazine