Aiming for multiple targets for Huntington’s disease therapies: a hopeful report from the Yang lab at UCLA
At Risk for Huntington's Disease
by Gene Veritas
2M ago
  This article is in commemoration of Huntington’s Disease Awareness Month (May).   One of the most impactful university labs focusing on Huntington’s disease, the X. William Yang Research Group at the University of California, Los Angeles (UCLA) employs a multi-pronged approach to investigating potential therapies for this deadly brain disorder.   Started in 2002 by X. William Yang, M.D., Ph.D., the lab has produced several key findings on HD, mainly through the study of genetically modified (i.e., transgenic) mice, engineered to carry the HD mutation and exhibit some of the di ..read more
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‘Striving for a cure’: highlights from the 19th Annual Huntington’s Disease Therapeutics Conference
At Risk for Huntington's Disease
by Gene Veritas
3M ago
  Progress towards effective treatments for Huntington’s disease relies on the collaboration of the HD-affected families’ collaboration with researchers exploring the frontiers of science.   The potentially pathbreaking findings featured at the recently completed 19th Annual HD Therapeutics Conference, sponsored by the nonprofit CHDI Foundation, Inc., led CHDI Chief Scientific Officer Robert Pacifici, Ph.D., to declare that the community will achieve therapies.   In this article I highlight the scientists’ work with a photo essay on their conference presentations and some of the ..read more
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Huntington’s disease community will 'get there' in search for therapies, CHDI chief scientist declares after ‘terrific’ conference
At Risk for Huntington's Disease
by Gene Veritas
4M ago
  After presiding over a “terrific” research conference, CHDI Foundation Chief Scientific Officer Robert Pacifici, Ph.D., declared that the Huntington’s disease community will “get there” in the search for long-awaited therapies.   Dr. Pacifici commented in an interview with me on March 1, after the CHDI-sponsored 19th Annual HD Therapeutics Conference, held in Palm Springs, CA, from February 26-29.   The CHDI Chief Scientific Officer (CSO) provided his optimistic assessment in referencing the featured presentation by David Altshuler, M.D., Ph.D., CSO of the Boston-based Vertex ..read more
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At CHDI conference, advocates inspire acceleration of quest for Huntington’s disease therapies
At Risk for Huntington's Disease
by Gene Veritas
4M ago
  With a record 420-plus participants, the 19th Annual Huntington’s Disease Therapeutics Conference got under way on February 26 with the aim of speeding the quest for therapies to slow, halt, or reverse the symptoms of this incurable disorder.   Sponsored by CHDI Foundation, Inc., the largest private funder of HD research, the event runs through February 29 at the Parker hotel in Palm Springs, CA, and will feature three days of scientific and clinical presentations.   “In recent years the quest for HD therapeutics that will make a real difference to affected families has accele ..read more
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Scientists interacting with Huntington’s disease patients in the quest for therapies
At Risk for Huntington's Disease
by Gene Veritas
5M ago
  In the quest for Huntington’s disease therapies, scientists have found key intellectual fuel for understanding the genetics of this fatal neurodegenerative disorder and developing therapies.   A brainstorming strategy became the trademark of the HD-focused Hereditary Disease Foundation (HDF), founded in 1974 by leading Los Angeles psychoanalyst and HD activist Milton Wexler as an offshoot of the Huntington’s Disease Society of America (HDSA).   Wexler organized multidisciplinary small workshops of scientists aimed at spontaneous discussion – as opposed to dry scientific presen ..read more
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New book by longtime advocate describes Milton Wexler’s incomparable contributions to Huntington’s disease research and beyond
At Risk for Huntington's Disease
by Gene Veritas
8M ago
  A new book portrays the largely unexplored personal and psychological context of the quest to understand and defeat Huntington’s disease: a biographical memoir of Milton Wexler (1908-2007), the founder of the Hereditary Disease Foundation (HDF) and key mover in the discovery of the HD gene.   In late 2022, Wexler’s daughter, historian Alice Wexler, published The Analyst: A Daughter’s Memoir (Columbia University Press). She is a longtime Huntington’s disease advocate and chronicler of the cause.   The Analyst adds unique dimensions to HD history, building on Alice’s groundbreak ..read more
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Adding to arsenal of movement disorder drugs approved by FDA, Neurocrine pledges to seek anxiously awaited therapies to slow progression of Huntington’s disease
At Risk for Huntington's Disease
by Gene Veritas
11M ago
  Neurocrine Biosciences, Inc., announced on August 18 that the U.S. Food and Drug Administration (FDA) has approved its drug INGREZZA to treat chorea, a debilitating movement disorder suffered by people with Huntington’s disease.   INGREZZA is the third FDA-approved HD chorea drug.   Like the two previously approved, similar drugs for chorea made by other drug companies, INGREZZA does not fulfill what the HD community of scientists, advocates, and affected families anxiously await: development and FDA approval of a drug to slow, halt, or reverse the progression of the disease ..read more
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Lessons from the Maui wildfires for human solidarity and the fight against Huntington’s and other diseases
At Risk for Huntington's Disease
by Gene Veritas
11M ago
  In late June – vacationing in the balmy Hawaiian town of Lahaina – my wife Regina and I snorkeled at Pacific Ocean coral reefs teeming with marvelous aquatic life, dined on succulent seafood, and recharged our emotional batteries after a difficult first half of 2023.   We had celebrated our 30th wedding anniversary by traveling to Hawaii for the first time, to the islands of Kona and Oahu, in March 2022. I had never expected to reach my early 60s healthy: I carry the deadly gene for Huntington’s disease, which took my mother at 68. Inspired by the spirit of aloha, we returned with ..read more
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After another critical Huntington’s disease clinical trial proves negative, a time to embrace self-care and caregiving
At Risk for Huntington's Disease
by Gene Veritas
1y ago
  A long-awaited clinical trial of a drug aimed at improving daily function  in the early stages of Huntington’s disease has produced negative results, Prilenia Therapeutics announced on April 25 at the American Academy of Neurology meeting in Boston.   According to Prilenia, the drug, pridopidine, failed to show improvement for trial participants on its primary and secondary measurements of symptoms (endpoints).   "Unfortunately, the failure of the PROOF-HD trial to meet its primary endpoint in preliminary analyses is a huge disappointment for the HD community,” Jody Corey ..read more
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#FightDiseaseNotWar
At Risk for Huntington's Disease
by Gene Veritas
1y ago
This holiday season – with the Ukraine conflict having raised fears of a World War III and hundreds of millions of people still lacking cures for neurological disorders, cancer, and thousands of other maladies – it’s time for people and their leaders everywhere to embrace a new global ethic: #FightDiseaseNotWar.   With the U.S. sadly far in the lead, nations spend trillions annually on their armed forces.   A Huntington’s disease gene expansion carrier who lost his mother to this devastating disease, I continue to watch HD brothers and sisters face enormous hurdles in their care – an ..read more
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