Although Nancy Wexler’s efforts were largely responsible for creating a genetic test to locate the Huntington’s gene, she refused to take the test herself saying “I will meet the devil…
Novartis and PTC Therapeutics have advanced their Huntington's disease candidate, votoplam, into Phase 3 development following promising mid-stage results. The decision reflects confidence in the program's potential while signaling caution…
As reported on MedicalXpress, a newly developed RNA-focused analytical method may improve the detection and characterization of repeat expansion disorders, a group of genetic conditions that includes Huntington’s disease, amyotrophic…
UniQure has announced plans to submit its groundbreaking Huntington's disease gene therapy, AMT-130, for regulatory approval in the United Kingdom later this year, marking a significant step forward for the…
As reported on PharmaBiz, Roche has secured CE mark approval for its Elecsys Neurofilament Light Chain (NfL) assay, a blood-based test designed to help detect neuroinflammation in adults with relapsing-remitting…
Researchers at Florida Atlantic University (FAU) have identified a cellular pathway that allows brain cells to share proteins and other toxic material. The study was recently published in Science Advance.…
Fight. I don’t think that those who aren’t in the Huntington’s Disease community understand what the word FIGHT truly means to those of us inside the community. We fight for…
As reported in Business Wire, Vico Therapeutics has initiated patient dosing in an expanded cohort of its Phase 1/2a clinical study evaluating VO659, an antisense oligonucleotide (ASO) designed to target…
As reported on PharmaBiz, Harness Therapeutics has selected HRN001 as its lead therapeutic candidate for Huntington’s disease (HD), marking a major milestone for the company as it prepares to transition…
An experimental gene therapy known as AMT-130 has become the first treatment to successfully slow the progression of Huntington’s disease, marking a potential breakthrough in the fight against neurodegenerative disorders.…
As reported by HD Buzz, a significant step forward for Huntington’s disease (HD) research, Roche has announced that the first patient has been dosed in its new gene therapy clinical…
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…
Editor's Note: We believe that patients are a key part of developing and leading the conversation in disease communities. Patient Worthy sometimes partners with reputable agencies that wish to speak…
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…
Your HTT gene encodes for the production of huntingtin, a protein that helps your neurons (nerve cells in your brain) function correctly. When mutations happen in this gene, the protein becomes…
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…
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