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‘Phenomenal’ trial results may lead to a treatment for Huntington’s disease, experts say

via the Washington Post: The discovery of a drug that may treat the fatal disease known as Huntington’s is being hailed as “historic” by Louise Vetter, president and CEO of the Huntington’s Disease Society of America, and “phenomenal” and “fantastically promising” by Huntington’s researchers, including the woman who discovered the genetic mutation that causes the disease. “I’m […]

Ionis Antisense Oligonuleotide Drug IONIS-HTTRX is Safe and Decreases Levels of Toxic Huntington’s Disease Protein in Cerebrospinal Fluid.

In a statement released today, Ionis Pharmaceuticals announced study results demonstrating for the first time that their drug (IONIS-HTTRX) lowered levels of the abnormal protein causing Huntington’s disease in cerebrospinal fluid, and that this approach was safe and well tolerated in humans. Link:   The IONIS-HTTRx Study: The IONIS-HTTRX trial enrolled 46 patients with […]