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Research Updates


HDF President Nancy Wexler is co-author on two major new studies of HD prevalence

Click below to read the full articles:

Evans SJ, Douglas I, Rawlins MD, Wexler NS, Tabrizi SJ, Smeeth L. "Prevalence of adult Huntington's disease in the UK based on diagnoses recorded in general practice records." J Neurol Neurosurg Psychiatry. 2013 Mar 29.

Douglas I, Evans S, Rawlins MD, Smeeth L, Tabrizi SJ, Wexler NS. "Juvenile Huntington's disease: a population-based study using the General Practice Research Database." BMJ Open. 2013 Apr 3;3(4).

Eureka!! Glorious news to start off the New Year!!! Read and enjoy!!!

The Hereditary Disease Foundation has supported this research from its first inception as an idea! We are very proud of the brilliant, dedicated, creative, persevering and all-around fabulous scientists who carried out this work with energy, enthusiasm, imagination and aplomb!

Congratulations, Joan, Leslie and William and all your magnificent and hard working group of investigators who put so much of their heart and soul into this work!!

Again, we are very proud!

Your Hereditary Disease Foundation family:
Nancy Wexler
Carl Johnson
Alice Wexler
The Scientific Advisory Board
The Board of Directors


JCB: IKK phosphorylates Huntingtin and targets it for
degradation by the proteasome and lysosome

Cell Press: Serines 13 and 16 Are Critical Determinants
of Full-Length Human Mutant Huntingtin Induced
Disease Pathogenesis in HD Mice

Click below to read recent press about these exciting findings:

JCB: IKK cuts both ways in Huntington’s disease

Press Association: 'Molecular switch' may halt disease

EurekAlert!: Small changes in protein chemistry play large role in Huntington's disease

The ALS Forum: Adorn That Amino End: Huntingtin Decorated for Destruction

ScienceDaily: Subtle Change Dramatically Reduces Pathogenic Potential of Huntington's Protein

ScienceBlog: UCLA scientists find molecular switch to prevent Huntington's disease in mice

BBC News: 'Switch' could block Huntington's

BusinessWeek: Scientists Find Molecular Switch Related to


The University of Massachusetts and Lundbeck Inc. Enter Research Collaboration to Explore Potential Targeted Therapy for Huntington's Disease

Gene silencing - a promising, revolutionary approach to curing Huntington's disease - gets a boost from a new academic-company partnership!
Neil Aronin, M.D., Professor of Medicine and Cell Biology, Department of Medicine and Graduate School of Biomedical Sciences, Chief, Division of Endocrinology and Metabolism, Co-Director, Neuro-therapeutics Institute, University of Massachusetts Medical School, shakes hands with Lundbeck Inc. to synergize the work.
Neil Aronin; Beverly L. Davidson, Ph.D., Roy J. Carver Professor in Internal Medicine, Professor in Neurology, and Physiology & Biophysics, Director, Gene Transfer Vector Core, Associate Director, The Iowa Center for Gene Therapy, Vice Chair for Research, Department of Internal Medicine, University of Iowa; Henry L. Paulson, M.D., Ph.D., Lucile Groff Professor, Department of Neurology, University of Michigan Health System; and X. William Yang, M.D., Ph.D., Associate Professor, Center for Neurobehavioral Genetics, Semel Institute for Neuroscience & Human Behavior, Department of Psychiatry & Biobehavioral Sciences, Brain Research Institute, David Geffen School of Medicine at UCLA - all silencing and animal model pioneers - have joined the 2011 Hereditary Disease Foundation Scientific Advisory Board.
Click here to read about this exciting collaboration.

First Treatment Approved for Chorea in HD Now Available in the U.S.
Xenazine* (tetrabenazine) Tablets, the first and only U.S. Food and Drug Administration (FDA) approved drug for the treatment of chorea associated with Huntington’s disease, is now available in the United States. Click here for more information about the availability of Xenazine (tetrabenazine) Tablets

* Xenazine is a registered trademark of Cambridge Laboratories (Ireland) Limited.

Cure Found for Huntington Disease in Mice Offers Hope for Treatment in Humans—VANCOUVER, B.C., June 16, 2006
Researchers at the Child and Family Research Institute's Centre for Molecular Medicine and Therapeutics (CMMT) have provided ground-breaking evidence for a cure for Huntington disease in a mouse offering hope that this disease can be relieved in humans.

Published today in Cell journal, Dr. Michael Hayden and colleagues discovered that by preventing the cleavage of the mutant huntingtin protein responsible for Huntington disease (HD) in a mouse model, the degenerative symptoms underlying the illness do not appear and the mouse displays normal brain function. This is the first time that a cure for HD in mice has been successfully achieved. [click here for full press release]

Research holds promise for Huntington’s treatment—Researchers at MIT and Harvard Medical School have identified a compound that interferes with the pathogenic effects of Huntington's disease, a discovery that could lead to development of a new treatment for the disease... FROM
[ Read Ruth Bodner's article here (PDF format) ]

Neural Transplants Provide Persistent Benefit In Patients With Huntingtonās Disease—Neuronal transplantation in Huntingtonās disease provides a period of improvement and stability of several years, according to an article published Online today (Monday February 27, 2006) by The Lancet Neurology.

Effect of fetal neural transplants in patients with Huntingtonās disease 6 years after surgery: a long-term follow-up study
Article: Anne-Catherine Bachoud-Lévi, Véronique Gaura, Pierre Brugires, Jean-Pascal Lefaucheur, Marie-Franoise Boissé, Patrick Maison, Sophie Baudic, Maria-Joao Ribeiro, Catherine Bourdet, Philippe Remy, Pierre Cesaro, Philippe Hantraye, Marc Peschanski

Drug Found To Reduce Involuntary Movements In Huntington's Disease—Tetrabenazine Update:

Study Finds Drug May Cut Down Involuntary Movements In Huntington's Disease Patients By 25 Percent - [.pdf
Drug aimed at Huntington's eases chorea, the disease's hallmark feature - [ .pdf ]
Study Published in Neurology Shows Tetrabenazine Significantly Improves Debilitating Movement Disorder Caused by Huntington's Disease [ .pdf ]
Prestwick drug looks good for Huntington's [

Venezuelan Kindreds Reveal Genetic and Environmental Factors Influence Onset of Huntingtonās Disease: New York, New York, March 2, 2004—The age at which Huntington’s disease starts is not solely determined by the Huntingtonās disease gene, as previously believed, but is strongly influenced by genetic and environmental factors, according to new findings from a team led by Columbia University Medical Center researcher and president of the Hereditary Disease Foundation Dr. Nancy Wexler.

Gene therapy may switch off Huntington's—March 13, 2003— HD Lighthouse—Using gene therapy to switch off genes instead of adding new ones could slow down or prevent the fatal brain disorder Huntington’s disease. The method, which exploits a mechanism called RNA interference, might also help treat a wide range of other inherited diseases.

Major Advances in Huntington's Disease Research Expected From Powerful Technology to Study Protein Structure—December 04, 2002—The Hereditary Disease Foundation (HDF) and Structural GenomiX, Inc. (SGX) today announced a collaboration agreement to investigate the structure of huntingtin, the protein responsible for Huntingtonās disease.

Huntington's Disease Study Offers New Hope—November 20, 2001—First hints that something can be done to defeat the inherited brain disorder called Huntington's disease are coming from experiments in fruit flies.

Hereditary Disease Foundation Scientists Find Possible Treatment For Huntington’s DiseaseOctober 22, 2001—UC Irvine Researchers Show for the First Time How Existing Therapies May Arrest and Prevent Nerve Damage in Incurable Genetic Brain Disorder.

Therapeutic Opportunities in Polyglutamine Disease Nature Medicine. April 2001. Research towards a therapeutic drug in many polyglutamine diseases with a focus on Huntington's Disease. Drs. Bob Hughes and Jim Olson are two of the many HD researchers who have been instrumental in the steady progress towards therapeutic interventions.

Treatment for Huntington's Disease Shows Promise LA Times. November 30, 2000. French researchers find that fetal brain cell transplants reversed the disease in three of five patients over a two year period. Also visit The Lancet, a fetal cell research website.

Green light to a cure Jellyfish’s eerie glow may be the key to unlocking Huntington’s Disease Newsday. October 3, 2000. The glow, they hope, will lead toward a cure for Huntington's disease

Jellyfish May Help Scientists Decipher Genetic Mysteries Wall Street Journal. September 12, 2000. Helps scientists tackle one of the most daunting challenges facing drug-hunting researchers: quickly turning the spate of new gene discoveries into innovative medicines.

Reversal of Neuropathology and Motor Dysfunction in a Conditional Model of Huntington’s Disease. Ai Yamamoto, Jose J. Lucas, & Rene Hen. Cell, March 31, 2000. FEATURING HDF SUPPORTED RESEARCH.

Reversing Neurodegeneration: A Promise Unfolds. Harry T. Orr & Huda Y. Zogbi. Cell, March 31, 2000.

Delaying the Onset of Huntington's in Mice. Anto van Dellen et. all. Nature, April 13, 2000.

Disease’s Surprise Data could lead to Huntington's treatment. FEATURING HDF SUPPORTED RESEARCH. Newsday Article, 5/18/2000.

Largest Huntington's Drug Study Ever

On the Trail of a Killer, They Discovered Hope. Los Angeles Times article from Sunday, November 14, 1999.

Crucial Turning Point in the Quest for a Cure for Huntington's DiseaseIn the August 8 issue of Cell, two scientific papers report a crucial breakthrough in the quest for a cure for Huntington's disease. Possibly the biggest news for someone at risk for Huntington's disease. August 7, 1997.


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