Nancy Wexler Receives Prestigious 2019 Double Helix Medal from Cold Spring Harbor Laboratory

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Nancy Wexler, HDF President, adds yet another incredibly well deserved honor to her long list of prestigious accolades! She was chosen to receive the 2019 Double Helix Medal for her extraordinary work leading to the discovery of the location of the gene that causes Huntington’s disease.

Learn more here about this wonderful honor!

Congratulations to Nancy for this wonderful recognition of your work to find treatments and cures for HD!!!

ON PBS NOW – “WOODY GUTHRIE ALL-STAR TRIBUTE CONCERT 1970” - Long-lost footage from concert benefiting what is now the HDF

Left to right: Arlo Guthrie, Joan Baez, Country Joe McDonald. Credit: Courtesy Hereditary Disease Foundation

Left to right: Arlo Guthrie, Joan Baez, Country Joe McDonald. Credit: Courtesy Hereditary Disease Foundation

We are thrilled to let you know that PBS stations around the country are airing the Woody Guthrie All Star Tribute Concert. This concert at the Hollywood Bowl was the very first fundraiser for what is now Hereditary Disease Foundation. You’ll see never before seen footage of this rare and historic event from 1970. 

As an added bonus, HDF will receive a portion of the proceeds from the sale of each DVD of this historic concert. Purchase here.

Woody Guthrie, America’s greatest folk singer, died of Huntington’s disease several years before this tribute concert was filmed. Over the past 50 years, we have made many significant discoveries about HD, and we are now celebrating the first gene therapy which promises to slow or even reverse HD in its tracks!

Woody Guthrie continues to inspire us. His music and memory live on forever. Don’t miss this amazing concert. Check your local listings for airtimes in your area!!!

Learn more about this exciting event on PBS . Read PBS’s press release.

“I come from an HD family.” Kate Miner, HDF Gala 2018

Kate Miner, of the band Miner, spoke at our October 22, 2018 Gala: “I come from an HD family. We’ve had Huntington's disease in our family’s genetic makeup our entire lives but we didn’t know about it until the summer of 2014. That was the year my mom’s sister Diane was diagnosed. This diagnosis had implications for my mom, my sisters and me…We were totally different people as we were trying to come to terms with this new reality…we felt lost at sea, we were in a place of uncertainty…we wanted to find a way back to yesterday.”

Watch her full remarks.

Listen to Kate and her husband Justin – “The Miners” - perform.

"We are on the cusp of groundbreaking discoveries!" Karen Newman, HDF Gala 2018

At the Hereditary DIsease Foundation October 22, 2018 Gala, Karen Newman, member of our Board of Directors and Gala Chair, spoke about the work of the Hereditary Disease Foundation: "This evening we celebrate the Hereditary Disease Foundation, an organization that has pioneered scientific research and discovery for 50 years. As a member of a family that has been affected by Huntington's disease, I know the profound impact that this foundation’s work has had on so many lives.”

Watch her full remarks.

Frank Gehry awards C. Frank Bennett the 2019 Leslie Gehry Brenner Prize for Innovation in Science, October 22, 2018

On October 22, 2018, Frank Gehry, member of the Hereditary Disease Foundation Board of Directors and Founding Trustee, presented the 2019 Leslie Gehry Brenner Prize for Innovation in Science to C. Frank Bennett of Ionis Pharmaceuticals. “A few years ago my young daughter passed away from ovarian cancer,” said Mr. Gehry, “We created a fund in her name – Leslie Gehry Brenner – and we give that award every year. The idea was to give it without any strings and see what happens. I love that idea – the freedom that it implies.”

Watch the full Award presentation.

The Magic of New Beginnings - News from the Hereditary Disease Foundation

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We’re excited about the beginning of a New Year and the promise 2019 holds for dramatic advances in HD research. Great things are happening…brilliant minds are at work. With your support, we will continue to push until we push HD out of the normal lifespan. Momentum begins with a moment. This is our magical moment…together we will make it happen for our families and for the future.

Read more in our January 2019 Newsletter.

"A new era for treating neurological diseases" HDF 2018 Symposium, Dr. C. Frank Bennett, Ionis

"A new era for treating neurological diseases" HDF 2018 Symposium, Dr. C. Frank Bennett, Ionis

The mood within the room was electric with excitement and hope as Dr. C. Frank Bennett, senior vice president for research and franchise leader for neurological programs at Ionis Pharmaceuticals, took the podium at the Hereditary Disease Foundation October 22, 2018 Gala. He gave a mesmerizing talk

about his work developing antisense therapeutics for neuromuscular and neurological diseases. His pioneering work brought us the world’s first approved treatment for spinal muscular atrophy (SMA). In 2005, Frank started a program to develop antisense therapies for Huntington’s disease. IONIS-HTTrx is designed to treat patients with HD and is the first and only drug to demonstrate reduction of the neurotoxic abnormal huntingtin protein, the underlying cause of HD, in patients. Ionis is partnering with Roche for phase 3 of the trial, which is set to begin in the U.S. and abroad soon!! This is a groundbreaking advance for the field of HD!

Click here to watch in full to Dr. Bennett’s October 22, 2018 Symposium Presentation.

BIG NEWS: Roche/Genentech Announce Site Locations for GENERATION HD1 Trial of RG6042

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Roche/Genentech announce the locations, including 20 sites in the U.S. and 6 in Canada, for their phase 3 study - called GENERATION HD1 - to test efficacy of the huntingtin-lowering therapy RG6042.

This study is open and currently recruiting! This will be a 2-year study in people with early diagnosed HD, involving monthly spinal injections to deliver RG6042 or a placebo, as well as clinical tests and scans to understand the effect of the drug on HD symptoms. Around 660 people age 25-65 will be recruited at approximately 90 sites across the world.

Read the full press release.

"This is a magical moment!" HDF 2018 Symposium - Dr. Shirley Tilghman, Princeton University

“This is a magical moment. This is a moment when discoveries are going to really translate into better medical practice,” said Dr. Shirley Tilghman.

A rapt audience listened intently as Dr. Shirley Tilghman, president emerita and professor of molecular biology and public affairs at Princeton University, spoke about how genes are organized in the genome and regulated during early development.  A member of the National Research Council’s committee that set the blueprint for the United States effort in the Human Genome Project, she was also one of the founding members of the National Advisory Council of the Human Genome Project for the National Institutes of Health.  Dr. Tilghman is a world-renowned leader in the field of molecular biology whose brilliant scientific career as a mammalian development geneticist has advanced our understanding of Huntington’s disease and other disorders.

Click here to watch in full to Dr. Tilghman’s October 22, 2018 Symposium Presentation.