Breaking News: Exciting Results of HDF-Supported Research Published in New England Journal of Medicine


“We are ecstatic by this very encouraging news!” exclaims Hereditary Disease Foundation (HDF) President Nancy Wexler.

The first human trial, which uses a novel approach to lower the level of the devastating huntingtin protein in the nervous system, is a success. The drug targeting the cause of Huntington’s disease proved safe and well tolerated.

The trial was led by Sarah Tabrizi, HDF Scientific Advisory Board Member, recipient of the 2017 Leslie Gehry Brenner Prize for Innovation in Science and Director of the University College of London (UCL) Huntington’s Disease Center. The study was sponsored by Ionis Pharmaceuticals, where HDF Scientific Advisory Board Member and 2018 Leslie Gehry Brenner Prize recipient C. Frank Bennett is senior vice president for research.

The top-level results were announced in December 2017, and now the full set of results of this breakthrough HDF-supported research are published in the prestigious New England Journal of Medicine accompanied by an editorial by Nancy Wexler and Kenneth Fischbeck of the National Institutes of Health (NIH) and HDF Scientific Advisory Board member. 

Nancy Wexler said, “We are excited that we will now be moving forward to a global Phase 3 trial sponsored by F. Hoffman-LaRoche, which licensed the medicine. HDF has supported the preclinical development of this promising therapy for the past decade.”

HDF-supported research is yielding great results!

Read the article online or click here for the PDF.

Read the editorial online or click here for the PDF.

View the supplementary appendix online or click here for the PDF.