For many years, scientists have known about toxic clumps forming in patients with Huntington’s disease (HD). However, until recently, scientists relied on examining post mortem brain samples of HD…
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…
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…
As reported in Biospace, Taysha Gene Therapies wants to eradicate devastating genetic epilepsies and central nervous system disorders rooted in a single genetic mutation. That’s going to take some innovative…
When children have problems at school, it is often linked to something bigger going on in another aspect of their life, whether that is at home or with friends. A…
by Lauren Taylor from In The Cloud Copy Huntington’s disease is an inherited disease that slowly causes the breakdown of the nerve cells of the brain, resulting in various disorders…
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…
HDSA 35 New Orleans: The 35th Annual HDSA Convention June 4th-6th, 2020 The 35th annual convention is tailored for Huntington's disease patients and their families. Join the HDSA for three…
Ionis Pharmaceuticals, in a partnership with Roche, has announced that the enrollment has been completed for the third phase of GENERATION HD1, a trial intended to study the safety and…
As previously published in Scientific American, slightly after her first birthday, Emma Larson lost the use of her legs and started experiencing difficulty crawling. She was diagnosed with spinal muscular…
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…
Research has recently been published in Nature Communications that shows the success of a new gene therapy in the treatment of Huntington's Disease. This therapy, an adeno-associated virus-based gene therapy, was tested…
By Jodee Redmond from In The Cloud Copy Massachusetts Institute of Technology (MIT) researchers have recently announced they have identified hundreds of genes that are required for neuron survival. The…
According to a story from Yahoo News, Dr. Nancy Wexler spent around 20 years in Venezuela conducting pivotal research on a deadly rare genetic disorder: Huntington's disease. On Lake Maracaibo,…
Antonio Maltese is a young man living in Virginia. His family has also been devastated by the hereditary rare illness called Huntington's disease. In fact, he was diagnosed with the…
NPR News recently featured an article announcing that scientists at Portland’s Casey Eye Institute have attempted to use CRISPR to edit a gene with the DNA still in place.…
Patient confidentiality is often seen as an absolute in the medical world. Many people believe that it should never be broken. Cases do exist that challenge this thought however, where…
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