It’s rare that medical headlines live up to their hype, but the results announced by Ionis Pharmaceuticals about its investigational Huntington disease drug are certainly a scientific breakthrough – if not yet a medical one. An “antisense oligonicleotide,” injected into the cerebrospinal fluid of people with the deadly Huntington gene, reduced the amount of the mutant huntington protein in their nervous systems.
What remains to be seen is whether larger clinical trials prove this scientific advancement translates into a viable new therapy for people who have this genetic condition. Dr. Vox chats with study investigator Dr. Edward Wild of University College London Institute of Neurology in this Medical Minute.
This Medical Minute originally aired on Sept. 12, 2017.
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