RSS Feed – HDBuzz

  • Pfizer Amaryllis trial ends in disappointment: no improvement in Huntington’s disease symptoms
    Pfizer has announced that the first-pass analysis of its 'Amaryllis' trial, testing a PDE-10 inhibitor drug, shows the drug did not meet its target of improving Huntington's disease symptoms. As a result, the open-label extension study will be stopped. This is not the news we'd been hoping for, but we've ... read more
    Source: HDBuzzPublished on 2016-12-16
  • Pfizer Amaryllis trial ends in disappointment: no improvement in Huntington’s disease symptoms
    Pfizer has announced that the first-pass analysis of its 'Amaryllis' trial, testing a PDE-10 inhibitor drug, shows the drug did not meet its target of improving Huntington's disease symptoms. As a result, the open-label extension study will be stopped. This is not the news we'd been hoping for, but we've ... read more
    Source: HDBuzzPublished on 2016-12-16
  • Important advances in next generation genome editing tools for Huntington’s Disease
    Recent days have seen a slew of news emerging regarding the use of something called genome editing as a potential therapy for genetic diseases like Huntington's Disease. These approaches, which include exotic sounding tools like zinc finger nucleases and CRISPR/Cas9, differ from more traditional ways reducing the impact of the ... read more
    Source: HDBuzzPublished on 2016-10-31
  • Important advances in next generation genome editing tools for Huntington’s Disease
    Recent days have seen a slew of news emerging regarding the use of something called genome editing as a potential therapy for genetic diseases like Huntington's Disease. These approaches, which include exotic sounding tools like zinc finger nucleases and CRISPR/Cas9, differ from more traditional ways reducing the impact of the ... read more
    Source: HDBuzzPublished on 2016-10-31
  • Sorry folks, the PRIDE-HD trial did NOT show that Pridopidine slows the progression of Huntington’s disease
    A recent press release from Teva Pharmaceuticals has the HD community excited, claiming "Pridopidine Demonstrates Slowing of Progression of Huntington Disease in PRIDE-HD Study". What's pridopidine, and what can we really say about HD progression in patients treated with it? A brief history of pridopidine in HDPridopidine is a drug ... read more
    Source: HDBuzzPublished on 2016-09-30
  • Sorry folks, the PRIDE-HD trial did NOT show that Pridopidine slows the progression of Huntington’s disease
    A recent press release from Teva Pharmaceuticals has the HD community excited, claiming "Pridopidine Demonstrates Slowing of Progression of Huntington Disease in PRIDE-HD Study". What's pridopidine, and what can we really say about HD progression in patients treated with it? A brief history of pridopidine in HDPridopidine is a drug ... read more
    Source: HDBuzzPublished on 2016-09-29
  • EuroBuzz 2016: The Euro-HD Network Meeting in The Hague
    Here's our roundup of all the science presented at the 2016 European Huntington's Disease Network biennial meeting - one of the biggest meetings of Huntingtons Disease families, scientists and care professionals.Our first science session is on recent breakthroughs understanding the biology of HD. (Sidebar: for the 1st time, science speakers ... read more
    Source: HDBuzzPublished on 2016-09-23
  • EuroBuzz 2016: The Euro-HD Network Meeting in The Hague
    Here's our roundup of all the science presented at the 2016 European Huntington's Disease Network biennial meeting - one of the biggest meetings of Huntingtons Disease families, scientists and care professionals.Our first science session is on recent breakthroughs understanding the biology of HD. (Sidebar: for the 1st time, science speakers ... read more
    Source: HDBuzzPublished on 2016-09-22
  • Ultra-rare mutations highlight the importance of the HD gene in brain development
    A relatively new technology called exome sequencing has identified a few families with novel mutations in their HD genes. These are different than the mutation that causes HD, but allow researchers to better understand the normal role of the HD gene.Normal HD gene functionThe mutation that causes HD instructs brain ... read more
    Source: HDBuzzPublished on 2016-08-29
  • Ultra-rare mutations highlight the importance of the HD gene in brain development
    A relatively new technology called exome sequencing has identified a few families with novel mutations in their HD genes. These are different than the mutation that causes HD, but allow researchers to better understand the normal role of the HD gene.Normal HD gene functionThe mutation that causes HD instructs brain ... read more
    Source: HDBuzzPublished on 2016-08-29
  • Deutetrabenazine for Huntington’s disease: a positive trial but the FDA says no? Not exactly…
    A scientific paper declares positive results for a trial of deutetrabenazine in Huntington's disease, but the headlines report the FDA has rejected the drug. Confusing stuff! The reality is positive overall for this new way of treating uncontrollable movements in HD, but patience will be needed to see where it ... read more
    Source: HDBuzzPublished on 2016-08-17
  • Deutetrabenazine for Huntington’s disease: a positive trial but the FDA says no? Not exactly…
    A scientific paper declares positive results for a trial of deutetrabenazine in Huntington's disease, but the headlines report the FDA has rejected the drug. Confusing stuff! The reality is positive overall for this new way of treating uncontrollable movements in HD, but patience will be needed to see where it ... read more
    Source: HDBuzzPublished on 2016-08-16
  • A support system gone wrong – glial cells contribute to HD symptoms
    A new piece of research has implicated a type of brain cells called glia in the development of HD symptoms. Regular mice injected with glial cells carrying the mutant huntingtin gene in the study developed symptoms associated with HD. Interestingly, this influence appears to work both ways — a mild ... read more
    Source: HDBuzzPublished on 2016-08-02
  • A support system gone wrong – glial cells contribute to HD symptoms
    A new piece of research has implicated a type of brain cells called glia in the development of HD symptoms. Regular mice injected with glial cells carrying the mutant huntingtin gene in the study developed symptoms associated with HD. Interestingly, this influence appears to work both ways — a mild ... read more
    Source: HDBuzzPublished on 2016-08-02
  • Slightly long CAG repeats are more common than we thought
    Everyone with Huntington's disease has inherited the same type of mutation from their mother or father, an extra-long repetitive stretch of the sequence C-A-G in their HD gene. But the length of the mutation varies between individuals, and longer repeats are associated with earlier onset of symptoms. A huge new ... read more
    Source: HDBuzzPublished on 2016-07-05
  • Slightly long CAG repeats are more common than we thought
    Everyone with Huntington's disease has inherited the same type of mutation from their mother or father, an extra-long repetitive stretch of the sequence C-A-G in their HD gene. But the length of the mutation varies between individuals, and longer repeats are associated with earlier onset of symptoms. A huge new ... read more
    Source: HDBuzzPublished on 2016-07-05
  • Early exposure to the HD protein may cause life-long symptoms
    When the ‘healthy’ HD gene functions as it should, one of its many jobs is in the development of normal embryos. Researchers have long assumed that the ‘mutant’ HD gene inherited by people with HD is still able to do this job, since HD patients develop normally and don’t show ... read more
    Source: HDBuzzPublished on 2016-06-28
  • Early exposure to the HD protein may cause life-long symptoms
    When the ‘healthy’ HD gene functions as it should, one of its many jobs is in the development of normal embryos. Researchers have long assumed that the ‘mutant’ HD gene inherited by people with HD is still able to do this job, since HD patients develop normally and don’t show ... read more
    Source: HDBuzzPublished on 2016-06-28
  • Electron beam snaps best images yet of Huntington’s disease protein
    Figuring out the shape of a protein can help scientists understand how it works and what goes wrong in disease. Huntingtin, the protein that causes Huntington's disease, has been an elusive target. A recent study using electron microscopes offers a striking glimpse of huntingtin, paving the way for future work.Seeing ... read more
    Source: HDBuzzPublished on 2016-06-21
  • Electron beam snaps best images yet of Huntington’s disease protein
    Figuring out the shape of a protein can help scientists understand how it works and what goes wrong in disease. Huntingtin, the protein that causes Huntington's disease, has been an elusive target. A recent study using electron microscopes offers a striking glimpse of huntingtin, paving the way for future work.Seeing ... read more
    Source: HDBuzzPublished on 2016-06-20
  • Planting trees together: The 2016 Huntington’s Disease Society of America Convention
    Nearly a thousand HD family members converged on Baltimore, Maryland for the 2016 Huntington’s Disease Society of America’s Annual Convention. We normally don’t write reports from patient and family conferences, but there was something special about the atmosphere of this year’s Convention that compelled us to pen a brief update.The ... read more
    Source: HDBuzzPublished on 2016-06-13
  • Planting trees together: The 2016 Huntington’s Disease Society of America Convention
    Nearly a thousand HD family members converged on Baltimore, Maryland for the 2016 Huntington’s Disease Society of America’s Annual Convention. We normally don’t write reports from patient and family conferences, but there was something special about the atmosphere of this year’s Convention that compelled us to pen a brief update.The ... read more
    Source: HDBuzzPublished on 2016-06-13
  • Huntingtin takes a trip: harmful proteins pass between brain cells
    Clumps of mutant huntingtin protein in brain cells are a hallmark of HD, and they build up slowly, occupying more and more cells over time. Recent research in mice shows that the harmful proteins can travel between neurons, setting off a chain reaction that leads to more sick cells and ... read more
    Source: HDBuzzPublished on 2016-06-06
  • Huntingtin takes a trip: harmful proteins pass between brain cells
    Clumps of mutant huntingtin protein in brain cells are a hallmark of HD, and they build up slowly, occupying more and more cells over time. Recent research in mice shows that the harmful proteins can travel between neurons, setting off a chain reaction that leads to more sick cells and ... read more
    Source: HDBuzzPublished on 2016-06-06
  • Important drug targets yielded by new genetic study of HD
    It’s a great mystery why different people with the same HD mutation sometimes develop symptoms at vastly different ages. Last year a huge genetic analysis gave us some interesting clues, and now, researchers are focusing in on the most promising results. A recent study shows that tiny changes within genes ... read more
    Source: HDBuzzPublished on 2016-05-02
  • Important drug targets yielded by new genetic study of HD
    It’s a great mystery why different people with the same HD mutation sometimes develop symptoms at vastly different ages. Last year a huge genetic analysis gave us some interesting clues, and now, researchers are focusing in on the most promising results. A recent study shows that tiny changes within genes ... read more
    Source: HDBuzzPublished on 2016-05-02
  • Thinking clearly about the earliest symptoms of HD and which brain regions control them
    Cognitive deficits, or difficulties thinking clearly, often appear well before the traditional clinical diagnosis of Huntington’s disease (HD). While many contend that the earliest cognitive deficits are caused by damage to the striatum – a structure deep in the brain known to be severely affected in HD – recent evidence ... read more
    Source: HDBuzzPublished on 2016-04-18
  • Thinking clearly about the earliest symptoms of HD and which brain regions control them
    Cognitive deficits, or difficulties thinking clearly, often appear well before the traditional clinical diagnosis of Huntington’s disease (HD). While many contend that the earliest cognitive deficits are caused by damage to the striatum – a structure deep in the brain known to be severely affected in HD – recent evidence ... read more
    Source: HDBuzzPublished on 2016-04-18
  • How NOT to write a news article about a clinical trial
    A recent article in the UK newspaper the Daily Telegraph has HD families very excited. The title, "First drug to reverse Huntington’s disease begins human trials", certainly sounds exciting! But what's really going on? HDBuzz is here to help us untangle hope from hype in the huntingtin lowering world.The typo ... read more
    Source: HDBuzzPublished on 2016-03-11
  • How NOT to write a news article about a clinical trial
    A recent article in the UK newspaper the Daily Telegraph has HD families very excited. The title, "First drug to reverse Huntington’s disease begins human trials", certainly sounds exciting! But what's really going on? HDBuzz is here to help us untangle hope from hype in the huntingtin lowering world.The typo ... read more
    Source: HDBuzzPublished on 2016-03-10
  • Huntington’s Disease Therapeutics Conference 2016 – day 2
    After an exciting day of science yesterday, day 2 saw updates on strategies to rid cells of the harmful mutant huntingtin protein and exciting reports on current and planned clinical trials.Wednesday morning - huntingtin lowering strategiesHuntingtin lowering or "gene silencing" may be the most exciting potential HD treatment strategy. Since ... read more
    Source: HDBuzzPublished on 2016-02-28
  • Huntington’s Disease Therapeutics Conference 2016 – day 3
    The third and final day of the 2016 Huntington's Disease Therapeutics Conference brings updates on cell replacement therapies including stem cells; and new ways to assess and model the progression of HD to help understand it and run crucial clinical trials.Thursday morning - replacing cells, restoring networksThe day in Palm ... read more
    Source: HDBuzzPublished on 2016-02-28
  • Could a new “jaw-dropping” breakthrough help treat Huntington’s Disease?
    Recent days have seen a torrent of news stories about a new technology, called CRISPR, which has been described as having potential application in Huntington's disease. Is this new technique as cool as it sounds? Possibly — but, as always, the truth is more complicated than the headlines suggest. The ... read more
    Source: HDBuzzPublished on 2016-02-25
  • Huntington’s Disease Therapeutics Conference 2016 – day 1
    If it's February, that means the the world's leading scientists are converging on Palm Springs for the annual HD therapeutics conference!Monday evening - updates from CHDI FoundationBefore the meeting officially starts, staff from the CHDI Foundation are giving updates about the exciting science happening there.Tom Vogt, vice president of Discovery ... read more
    Source: HDBuzzPublished on 2016-02-25
  • Harnessing the power of viruses to treat Huntington’s disease
    New therapies for disorders like Huntington’s disease are on the way, but getting the drugs to enter brain cells can be a major challenge. A group of scientists has redesigned and tested a harmless virus that can efficiently deliver a 'gene silencing' message throughout the brain in mice, much further ... read more
    Source: HDBuzzPublished on 2016-02-03
  • Through a Broader Lens: Looking at Non-Motor Symptoms in HD
    Common depictions of HD emphasizing only its movement symptoms paint an incomplete picture of the real disease. HD causes both motor and non-motor symptoms that, together, affect the entire body. Now, scientists are using a broader lens to explore this full set of HD symptoms and determine how symptoms might ... read more
    Source: HDBuzzPublished on 2016-01-25
  • Looking Past the Spin: Results from a Clinical Trial of Cysteamine
    In early December, Raptor Pharmaceuticals released clinical trial results evaluating a drug called cysteamine in Huntington’s disease. News headlines about this trial are heavy on media spin, and so HDBuzz is here to break down what these new results really mean for the Huntington's community.Clinical Trials and Pomegranates?Though it might ... read more
    Source: HDBuzzPublished on 2015-12-18
  • A recycled drug gives us new insight in to HD
    Researchers have found a connection between HD and an energy-regulating protein called PPAR-delta. Giving PPAR-delta a boost with an existing drug was protective in HD cells and mice, but we’ll likely need to research and test it further before it can go to the HD clinic. Repurposing therapiesSometimes a promising ... read more
    Source: HDBuzzPublished on 2015-12-15
  • Through the eyes of a friend: changes in mood and behavior in early HD
    The family and friends of individuals with HD often tell doctors that they began to notice changes in behavior long before a diagnosis was made. To better understand these early signs, researchers analyzed a psychological questionnaire filled out yearly for a decade by thousands of HD mutation carriers and their ... read more
    Source: HDBuzzPublished on 2015-12-01
  • A multiple sclerosis drug that works for Huntington’s disease: the real deal or too good to be true?
    Thinking problems in Huntington’s disease take a huge toll from early in the disease. Now, new work suggests that a drug already approved by the FDA to treat another brain disease – multiple sclerosis – may stave off these problems in HD mice. Could these results be real, or are ... read more
    Source: HDBuzzPublished on 2015-11-23
  • Mapping the anatomy of HD: a whole-brain history
    Though many scientists have focused on damage to a part of the brain called the striatum as a source of HD symptoms, this is a narrow picture of what changes in the brain during HD. A new book provides a summary of many research techniques over a hundred years that ... read more
    Source: HDBuzzPublished on 2015-11-04
  • Liftoff: First humans treated with gene silencing drugs for HD!
    Today brings news that the first Huntington's Disease patients have been successfully dosed with gene silencing drugs targeting the HD gene. These brave volunteers are the first HD patients to ever be treated with drugs designed to attack HD at its root cause, a treatment approach with huge potential. What ... read more
    Source: HDBuzzPublished on 2015-10-22
  • HDBuzz primer: Clinical trial designs and phases
    The last few years have been full of announcements about the results of clinical trials for HD drugs, but it can be surprisingly hard to understand what these results actually mean. What seems like a simple question – did a drug work or not – turns out to be more ... read more
    Source: HDBuzzPublished on 2015-10-09
  • A Few Bad Seeds: Using Brain Fluid to Grow Clumps in Brain Cells
    It’s like gardening gone wrong: scientists can sprinkle Huntington’s protein on the outside of laboratory-grown brain cells and make sticky, potentially harmful protein clumps grow inside the cells. Now, new research showing that human brain fluid does the same thing could help us monitor Huntington's disease.Huntington’s disease is caused by ... read more
    Source: HDBuzzPublished on 2015-09-14
  • New tool to measure outcomes in Huntington’s Disease clinical trials
    When patients participate in clinical trials, there needs to be some type of readout to determine whether the new treatment worked. It’s important to know two key things: What to measure and how to measure it. In the case of HD, these obstacles have vexed scientists and doctors for years. ... read more
    Source: HDBuzzPublished on 2015-09-03
  • Huge study reveals new ‘genetic modifiers’ of Huntington’s disease
    Even though every patient with Huntington's Disease has a mutation in the same gene, the age at which HD patients develop symptoms varies widely. A global consortium of HD researchers has just published a landmark study of genetic differences between people that might explain some of that variability, producing some ... read more
    Source: HDBuzzPublished on 2015-07-31
  • Would ewe believe it? GM1, sheep and Huntington’s disease
    It takes a lot of research to decide whether a new treatment is ready to test in humans. A few years ago, we reported that a 'brain fat' called GM1 is reduced in HD, and replacement therapy showed potential in mice. Despite rumors you may have heard, it’s not ready ... read more
    Source: HDBuzzPublished on 2015-07-28