https://uniqure.gcs-web.com/news-releases/news-release-details/uniqure-announces-submission-biologics-license-application

CBER (Center for Biologics Evaluation and Research) and CDER (Center for Drug Evaluation and Research) are two major centers within the U.S. Food and Drug Administration, each responsible for regulating different types of medical products: CBER: Focus: Regulates biologics—products derived from living organisms. Products regulated: Vaccines, blood and blood products, gene therapies, cellular therapies, allergenic extracts, and some medical devices related to these products. Ex

There are so many things that people in the Huntington’s Community never talk about. Especially the Caregivers or Care Partners (whatever your choice of description may be.) You think you know how hard this journey into Caregiving is going to be, but until you actually live it, you have no clue. It’s easy for those on the outside looking in to give suggestions that they think are helpful but really aren’t. One of the biggest things a Caregiver won’t tell you about is the guil

Ferrer and Prilenia are pleased to update you that the first participant has been randomized in the PRECISE-HD confirmatory Phase 3 study of pridopidine, an investigational medicinal product taken as an oral capsule twice daily, in Huntington’s disease (HD). This milestone marks the start of participant enrolment, with several US sites now active and additional sites in Canada, the EU and the UK set to follow later this year. PRECISE-HD is designed to further evaluate the eff

Because of an amazing private donor, we are able to offer 10 full scholarships to attend Help 4 HD's annual Symposium for first-time attendees. Restrictions apply; please read carefully before applying. https://www.help4hd.org/events-1/full-scholarships-to-help-4-hds-annual-symposium-1

Is It Better to Know What Your Partner’s Future Is? When your loved one is at risk for Huntington’s disease, the question of knowing the future takes on a new level of complexity. As a partner, you may wonder if learning your loved one’s genetic status will help you both prepare, or if it will cast a shadow over your relationship. Is it better to know what the future holds, or to live in uncertainty together? The Power of Shared Knowledge For many couples, having answers brin

At Help 4 HD International, we believe the people living with Huntington's disease — and the families who love and care for them — should have a real seat at the table when decisions get made about treatments. That's why we created a survey called Your Preferences and Perspectives on Potential Therapies, and it's why we're so proud to share our whitepaper built from your answers. More than 200 of you took the time to tell us what matters most. Caregivers, people living with H

We are thrilled to hear that Skyhawk Therapeutics has announced the expansion of its global pivotal FALCON-HD trial for SKY-0515 into the United States, Canada, and the United Kingdom! With more than ten countries and over twenty sites now involved, and over 175 patients enrolled, this milestone marks a major leap forward in the fight against Huntington’s disease. The Phase 1/2 study for SKY-0515 is fully enrolled and has shown encouraging safety and efficacy results, includi

PRECISE-HD (Pridopidine Phase 3 Study to Establish Clinical Impact and Safety in Huntington’s Disease (HD)) is designed to further evaluate the efficacy and safety of pridopidine, an oral investigational drug, on disease progression, functional capacity, motor function, cognition, speech and Quality of life -- Focused on generating the data to further support regulatory evaluation, PRECISE-HD will be undertaken in up to 75 sites globally, including the US, EU, UK and Canada.

Teva Pharmaceuticals’ Austedo (deutetrabenazine) and its extended-release formulation, Austedo XR — approved to treat chorea, or involuntary movements, in people with Huntington’s disease — have functional and social benefits for both patients and their caregivers. That’s according to real-world data presented today by Debbi Fox-Davis, Teva‘s director of patient engagement and advocacy, and Hannah Roth, Teva’s medical science liaison, at the 41st Huntington’s Disease Society

My name is Katie Jackson, and my journey with HD is both professional and deeply personal. My husband was diagnosed with HD at just 26. For 14 grueling years, I was his caregiver as he bravely battled this relentless disease, until I lost him at the age of 41. Now, six years later, I am once again staring down the barrel of this nightmare. My children are reaching the age where people are often considering testing, and after 20 years of fighting for them—20 years with no ther

"Huntington’s disease (HD) is a neurological disorder that can impact an individual’s physical, mental and emotional functioning. About 90% of individuals with HD will develop chorea, the involuntary movements associated with HD, and we know its symptoms profoundly impact not only the individual, but also their family members and care partners. New research presented at Advanced Therapeutics in Movement & Related Disorders (ATMRD) Congress highlights the significant impact

Raising Huntington’s Disease Awareness: Beyond the Month of May As May draws to a close and Huntington’s Disease (HD) Awareness Month ends, advocates are urging the public to continue the fight for awareness and support year-round. For those who do not know, Huntington’s Disease—a rare, inherited neurodegenerative disorder—affects thousands of families across the United States, yet remains widely misunderstood. Organizations like Help 4 HD International are leading the charge

It has been two years since I lost my dad to Huntington’s Disease. Living without him has been a profound journey, filled with many highs, lows, and everything in between. It would take an entire series to capture it all accurately. After he died, we began referring to that time as “The Year of Firsts.” The first Father’s Day, the first volleyball season, the first family photo without him. During that year, I remember feeling utterly exhausted, sleeping more than I ever had

“I don’t have hope….” My Huntington’s Disease Warrior blurted that out recently. We had been invited by a friend of mine to speak to a group of college students about Huntington’s Disease. I had asked Nathan if he wanted to go or if he wanted me to do this on my own (I gave him the choice all the way up to the time to go). He wanted to go and to give awareness to HD just as he’s always done. He was fine on the way, fine just before everything started….so imagine my shock whe