Powerful new approach helps understand molecular alterations in neurological disease

 CREDIT: BAYLOR COLLEGE OF MEDICINE

CREDIT: BAYLOR COLLEGE OF MEDICINE

Neurological diseases are typically associated with a multitude of molecular changes. But out of these thousands of changes in gene expression, which ones are actually driving the disease? Read full story.

Funding for this work was provided, in part, by a grant from the Hereditary Disease Foundation.

SAVE THE DATE - Jackie's Walk 4 HD, October 14, 2018

Jackie's Walk for HD 2018 will be held October 14th, at Glen Island Park in New Rochelle, NY with the Hereditary Disease Foundation (HDF). HDF was the first organization funding research and continues to inspire innovation in the field of Huntington's disease research. We are so happy to run this event as part of HDF’s 50th Anniversary celebration.

We hope that you will join us in this day of fun and celebration as the latest research for HD is so promising!

Click for more details.

Pope Francis champions Huntington's disease, by HDF President Nancy Wexler

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"From the moment I first visited Venezuela in 1979, which has the world’s highest prevalence of Huntington’s disease, I was moved and motivated to help and improve the lives of patients with Huntington’s disease, their families and their communities." 

Read full letter.

Washington Post features HDF President Nancy Wexler and CEO Meghan Donaldson

Pope Francis caresses a sick woman during an audience with Huntington's disease sufferers and their families on May 18, 2017. (Andrew Medichini/AP)

“I'm ecstatic,” said Nancy Wexler, who in 1993 identified the mutation. “Huntington's is horrible, one of the worst diseases known to mankind, and certain death. . . . We know it’s a bad gene, making a bad protein, that makes people sick, that kills your brain cells. Anything that could impact that, we knew that that could be a cure.”

Read the full story here.

Biggest news in Huntington's disease research since the identification of the HD gene!

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The Hereditary Disease Foundation congratulates Dr. Sarah Tabrizi, director of the University College London's Huntington Centre and the global lead investigator on the study, and her team on their breakthrough discovery. Congratulations also to C. Frank Bennett, senior vice president of research at Ionis Pharmaceuticals and longtime Scientific Advisory Board of the Hereditary Disease Foundation.

To read about this clinical trial, visit http://www.bbc.com/news/health-42308341.

Dr. Nancy Wexler, President of Hereditary Disease Foundation, said, "I'm ecstatic to see these promising positive results! If this therapy works to prevent or reverse HD, for the first time we will have something to immediately give people when they have a genetic test and discover that they are destined to die in the most catastrophic way."

This extraordinary and exciting breakthrough brings hope to the thousands of Huntington's disease patients and families worl dwide. This study will have an amazing impact on the HD community!

On November 6, the Hereditary Disease Foundation held a Symposium and Gala to launch our 50th Anniversary celebration. Sarah Tabrizi presented at the Symposium the latest from the "first in human" drug trial which "silences" - or turns down - the HD gene. Sarah also is the recipient of the Hereditary Disease Foundation's 2017 Leslie Gehry Brenner Prize for Innovation in Science for her research to discover treatments to prevent or reverse HD. We couldn't be prouder!

Sarah Tabrizi and Frank Bennett have been a part of the Hereditary Disease Foundation research family for many years. We are thrilled to see this research advance to such a remarkable point! A cure for the Huntington's disease is indeed within our reach.

Sarah Tabrizi's presentation about the Ionis clinical trial, November 6, 2017

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Sarah Tabrizi presented the latest from the “first in human” drug trial which “silences” – or turns down – the HD gene at the Hereditary Disease Foundation's 50th Anniversary Symposium and Gala on November 6, 2017.

Watch her presentation here.

Sarah also received the 2018 Leslie Gehry Brenner Prize for Innovation in Science that evening.