As discussed in Part I of this article, a recent HDBUZZ publication featured a news item stating that due to several severe adverse events occurring during the initial AMT-130 trial…
When we ‘do the math’ it adds up to the novel AMT-130 gene therapy receiving positive results from uniQure’s clinical trial investigating a small group (N=4) of individuals who were…
Have you ever thought about walking to raise funds and awareness? Well, at the Twin Cities Team Hope Walk, you can do just that! According to Kare11, this event…
It’s no secret that teachers can make a huge impact on the students in their classrooms. And that’s just what Judy Griffin did for her student Ashley. When Judy started…
In the United States, Orphan Drug designation is granted to drugs or biologics intended to treat, prevent, or diagnose rare diseases or conditions. A rare disease is one affecting under…
It’s finally May – and you know what that means! That’s right, this month is Huntington’s disease (HD) Awareness Month. The theme for this month’s HD Awareness Month is #LetsTalkAboutHD.…
When it comes to finding treatments for diseases, whether they're common or rare, the process doesn't always start from scratch. In many cases, existing drugs are taken and studied in…
HTT gene mutations cause Huntington’s disease, a rare and inherited neurological condition. Those with a family history of Huntington’s disease may choose to undergo genetic testing to learn more, though…
According to a recent press release from uniQure, the first round of observations from a trial investigating AMT-130 for Huntington's disease (HD) have been released. As of now, four patients…
Welcome to Study of the Week from Patient Worthy. In this segment, we select a study we posted about from the previous week that we think is of particular interest…
For many patients, symptoms of Huntington’s disease appear within midlife: between the 30s and 40s. This progressive disorder causes brain cell death, leading to a loss of physical and mental…
In the United States, Fast Track designation is granted to help expedite drug development and review for serious, rare, or life-threatening conditions. To receive Fast Track designation, the treatment must…
Patient Worthy signed up to cover several sessions at the Huntington's Disease Society of America's (HDSA) 36th Annual Convention, which was held virtually this year. This event features informative presentations…
Patient Worthy signed up to cover several sessions at the Huntington's Disease Society of America's (HDSA) 36th Annual Convention, which was held virtually this year. This event features informative presentations…
Patient Worthy signed up to cover several sessions at the Huntington's Disease Society of America's (HDSA) 36th Annual Convention, which was held virtually this year. This event features informative presentations…
For years, researchers have been evaluating pridopidine, developed by Prilenia Therapeutics ("Prilenia"), as a potential therapy for patients with neurodegenerative conditions. As reported in HD Buzz, new research has highlighted…
In the last decade, scientists have made great strides in uncovering details of the underlying causes of neurodegenerative diseases. It is only a matter of time that treatment and prevention…
According to a story from The Philadelphia Inquirer, 25 year old Gabby McNary isn't taking any risks when it comes to the coronavirus/COVID-19 pandemic. Philadelphia was hit hard by the…
uniQure has recently began their Phase I/II clinical trial of AMT-130, a gene therapy for the treatment of Huntington's disease. Two patients are included so far, with one being given…
A two-part series of “The Gene”, a PBS documentary, will air on April 14, 2020. The film, by Ken Burns, is based on a book with the same title written…
According to a recent article published in Pharmaceutical-Technology, uniQure’s gene therapy AMT-130 may lower levels of the huntingtin protein that causes the disorder. An abnormal gene is responsible for damage…
Current treatment for Huntington's disease only relieves symptoms, allowing for the progression of the disease to continue. UniQure, a company that works with gene therapies, aims to change this. They…
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Stéphanie Tomé is an investigator at the Sorbonne Université in Paris, France. A recent article in PacBio describes Tomé’s research into a disease that becomes progressively worse with each generation.…
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It's common knowledge among researchers who study diseases called toxic proteinopathies that this group of disorders are the result of misfolded proteins that reside in cells. Certain proteins fail to…
It's common knowledge among researchers who study diseases called toxic proteinopathies, that this grou of disorders are the result of misfolded proteins that reside in cells. Certain proteins fail to…
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