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…
My name is Antonio Maltese and I am a 22 year old senior Political Science major concentrating in International Relations and minoring in German at Virginia Commonwealth University. Back in…
Precision mNedicine is the latest craze in rare disease research. Thankfully for patients, it is completely, 100% centered on improving the quality of their care. Unlike most typical healthcare practices,…
Check out part one of Seth's story here. Seth had set a google alert for his hometown, Natick, to stay up to date in his home's local news. One day,…
When Seth was twelve years old, he and the rest of his family began to realize something was wrong with his mother. Seth can remember conversing with her pleasantly, and…
According to an article originally published by HDBuzz, a number of exciting advances have been made in the development of treatments that hope to treat thousands of potential Huntington's disease…
Is there a genetic condition in your family? Are you concerned about the potential for passing this on to your children one day? Do you know if you are a…
New pig models of Huntington’s disease have been developed, and they are expected to have several benefits compared to existing rodent models of the condition. Read the source article here…
Scientists in California at Salk Institute for Biological Studies are currently developing a drug that can mimic the effects in the body usually obtained from exercise, reports the National Post.…
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