You might have heard of Orphan Drug designation - and maybe even Rare Pediatric Disease designation. But have you heard of Regenerative Medicine Advanced Therapy (RMAT) designation? Described in Section…
The month of May is Huntington's Disease Awareness Month, in which a special effort is made to improve awareness about this devastating rare disease. This year, the Huntington's Disease Society…
For years, scientists have been working on a completely new concept that generates new neurons in mice with Huntington's disease. They were able to demonstrate that the new cells could…
Currently, there are no FDA-approved treatments designed for cognitive impairment related to Huntington’s disease. However, to improve the lives of those affected, additional research and therapeutic development are urgently…
Chorea, a characteristic manifestation of Huntington's disease, refers to uncontrolled "dance-like" movements in the fingers, feet, face, or torso. These movements may worsen in times of heightened emotion or…
uniQure, a leading gene therapy company, recently shared an update on a Phase 1/2 clinical study evaluating AMT-130 for Huntington’s disease. The safety and proof-of-concept study enrolled 26 participants with…
According to the National Institute of Neurological Disorders and Stroke, chorea is defined as involuntary movements that are caused by a neurological disorder. It is one of a group of…
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…
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…
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…
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…
Sign Up With a Patient Worthy Account and Share Your Rare Story
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
