RSS Feed – HDBuzz

  • A step forward for gene editing: CRISPR-Cas9 and HD
    CRISPR-Cas9 is an experimental gene editing technique used to make precise changes in DNA. For the first time, scientists have used this approach to attack the Huntington's Disease mutation in the brain cells of a mouse. Other researchers are refining CRISPR-Cas9 to be more efficient, specific, and safe. It’s still ... read more
    Source: HDBuzzPublished on 2017-07-26
  • Update confirms Huntington’s disease ‘gene silencing’ trial on track
    Ionis Pharmaceuticals launched the first ever trial of a huntingtin-lowering drug – sometimes called a 'gene silencing drug' – in late 2015. In a significant update, the company has announced two important milestones: the trial is now fully recruited, and an 'open-label extension' will be activated for the volunteers in ... read more
    Source: HDBuzzPublished on 2017-06-22
  • New study reveals a potential HD biomarker
    What if a blood test could provide information about the status and course of HD? This is the premise of seeking HD biomarkers; they may someday help guide treatment decisions and predict how symptoms will change. A team of researchers spanning multiple countries recently analyzed blood, brain images, and clinical ... read more
    Source: HDBuzzPublished on 2017-06-07
  • Has a “wonder drug” for dementia been discovered? (Spoiler alert: no.)
    If media reports of a "wonder-drug" that could "stop all neurodegenerative brain diseases, including dementia” seem too good to be true, that's because they are. The truth behind the headlines is that researchers tested thousands of already-licensed drugs in worms, and a couple that went on to show beneficial effects ... read more
    Source: HDBuzzPublished on 2017-05-07
  • Huntington’s Disease Therapeutics Conference 2017 – Day 3
    The final day of the 2017 Huntington's Disease Therapeutics conference brought updates on research to better understand how HD affects brains, and breakthroughs in using brain cells to understand and treat HD.Connections in the brainAndrew Leuchter from UCLA works on ways to study the brain's electrical activity to monitor HD. ... read more
    Source: HDBuzzPublished on 2017-04-30
  • Huntington’s Disease Therapeutics Conference 2017 – Day 2
    Day two of the conference looks at some of the most promising approaches to fighting Huntington's disease.Huntingtin lowering therapiesExciting session planned for this morning, as the conference discusses "Huntingtin lowering" approaches to treating HD.The first talk is from HDBuzz's own Ed Wild (University College London), who is interested in developing ... read more
    Source: HDBuzzPublished on 2017-04-29
  • Huntington’s Disease Therapeutics Conference 2017 – Day 1
    Hi everyone! It's the annual Huntington's Disease Therapeutics Conference in Malta. Around 350 scientists from round the world all working on HD are gathered here to discuss the latest research developments. We're reporting live via twitter and aggregating our updates into daily articles.Big news from big dataThe first speaker is ... read more
    Source: HDBuzzPublished on 2017-04-27
  • Enemy at the gates – huntingtin disrupts nuclear transport
    Exciting new studies provide evidence that a particular kind of cellular trafficking goes awry in Huntington's Disease. Specifically, researchers have learned that traffic in and out of the cells control center - the nucleus - breaks down in HD. These findings open up new avenues for HD research.Cellular border controlAt ... read more
    Source: HDBuzzPublished on 2017-04-21
  • FDA approves a new drug for symptoms of Huntington’s disease
    Significant news for the Huntington's disease community this week, as the USA's drug regulator, the FDA, has formally approved Austedo, also known as deutetrabenazine, for prescribing in HD. This modified form of tetrabenazine helps control chorea, the jerky movements often found in HD patients, but is taken twice rather than ... read more
    Source: HDBuzzPublished on 2017-04-05
  • Sheep leading the flock: metabolism and biomarkers in HD
    Beyond affecting movement, mood, and thinking, HD involves a complex set of changes to the body that can be difficult to predict in different individuals. Recently, researchers were able to identify consistent early changes in metabolism by studying a group of HD sheep. This large animal model is helping scientists ... read more
    Source: HDBuzzPublished on 2017-03-27
  • Weeding out the truth: can cannabis improve Huntington’s?
    Cannabis, or medical marijuana, has been touted as a treatment for lots of conditions, and Huntington’s disease is no exception. Whenever it hits the news there’s a lot of interest, and recently cannabis found the spotlight again with videos claiming it can reverse the nerve cell damage in Huntington’s disease. ... read more
    Source: HDBuzzPublished on 2017-02-24
  • Weeding out the truth: can cannabis improve Huntington’s?
    Cannabis, or medical marijuana, has been touted as a treatment for lots of conditions, and Huntington’s disease is no exception. Whenever it hits the news there’s a lot of interest, and recently cannabis found the spotlight again with videos claiming it can reverse the nerve cell damage in Huntington’s disease. ... read more
    Source: HDBuzzPublished on 2017-02-24
  • Huntingtin grabs a hammer: DNA repair in HD
    DNA damage is a hot topic in HD – and new research offers an intriguing explanation. Canadian researchers have uncovered a potential role for huntingtin in the repair of DNA. They speculate that the normal protein is recruited to the nucleus to provide a supporting scaffold for a construction crew ... read more
    Source: HDBuzzPublished on 2017-02-22
  • Huntingtin grabs a hammer: DNA repair in HD
    DNA damage is a hot topic in HD – and new research offers an intriguing explanation. Canadian researchers have uncovered a potential role for huntingtin in the repair of DNA. They speculate that the normal protein is recruited to the nucleus to provide a supporting scaffold for a construction crew ... read more
    Source: HDBuzzPublished on 2017-02-22
  • The benefits of migration, highlighted in Huntington’s disease
    Huntingtin, the protein responsible for Huntington’s disease, is fundamentally important for fetuses to develop in the womb, but we don’t know yet exactly what part it plays in this intricate process. Normally, neurons start life deep within the developing brain, migrate out to the surface and then make a network ... read more
    Source: HDBuzzPublished on 2017-02-10
  • The benefits of migration, highlighted in Huntington’s disease
    Huntingtin, the protein responsible for Huntington’s disease, is fundamentally important for fetuses to develop in the womb, but we don’t know yet exactly what part it plays in this intricate process. Normally, neurons start life deep within the developing brain, migrate out to the surface and then make a network ... read more
    Source: HDBuzzPublished on 2017-02-10
  • Treasure your exceptions – using outliers to understand Huntington’s disease onset and progression
    Researchers have long known that patients with a greater number of CAG repeats in their HD gene tend to display motor symptoms sooner than those with fewer CAG repeats. However, this relationship is not perfect. Sometimes HD patients display symptoms at a drastically different time than expected based on the ... read more
    Source: HDBuzzPublished on 2017-01-12
  • Treasure your exceptions – using outliers to understand Huntington’s disease onset and progression
    Researchers have long known that patients with a greater number of CAG repeats in their HD gene tend to display motor symptoms sooner than those with fewer CAG repeats. However, this relationship is not perfect. Sometimes HD patients display symptoms at a drastically different time than expected based on the ... read more
    Source: HDBuzzPublished on 2017-01-12
  • A powerful message: does a toxic RNA message molecule cause harm in Huntington’s disease?
    Researchers have long believed that the Huntington's disease gene causes problems by telling cells to make a harmful protein. Intriguing new animal work from researchers in Spain suggests we might want to look at more than one suspect to completely fix the problems caused by the HD mutation.The central dogma ... read more
    Source: HDBuzzPublished on 2016-12-30
  • A powerful message: does a toxic RNA message molecule cause harm in Huntington’s disease?
    Researchers have long believed that the Huntington's disease gene causes problems by telling cells to make a harmful protein. Intriguing new animal work from researchers in Spain suggests we might want to look at more than one suspect to completely fix the problems caused by the HD mutation.The central dogma ... read more
    Source: HDBuzzPublished on 2016-12-23
  • 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