Clinical Edge

Summaries of Must-Read Clinical Literature, Guidelines, and FDA Actions

Genetic Therapy Reduces Key Protein in Huntington’s Disease

Key clinical point: A genetic therapy reduced levels of a harmful protein in the cerebrospinal fluid of patients with Huntington’s disease.

Major finding: Mean reductions of between 20% and 42% of cerebrospinal fluid concentrations of mutant huntingtin with treatment versus an increase of 10% in the placebo arm.

Study details: A randomized, double-blinded, multiple-ascending-dose, placebo-controlled trial enrolling 46 patients with early Huntington’s disease in three countries.

Disclosures: The study was funded by Ionis Pharmaceuticals and F. Hoffmann–La Roche, and most of the authors, including Dr. Tabrizi, reported financial relationships with one or both entities.


Tabrizi SJ et al. N Eng J Med. 2019:380;2307-16.