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

Email us, if you would like to participate! Katrina@Help4HD.org To submit a video on any of the above topics, please reach out! We can interview you, or you can send in a video. Here is a link to set up a time for video recording: Registration Form | Help 4 HD Internatio Click the link below to receive a #Help4HDSwag box for HD Awareness Month 2026 To receive a Help 4 HD ~ HD Awareness box, please click here: Registration Form | Help 4 HD Internatio

Help 4 HD will be in Baltimore for our first in-person HIPE of 2026. Our schedule is packed full of content sure to fulfill everyone's interests whether that be chorea management, upcoming and current trials, community stories, what to expect during a clinical trial, or a panel of experts ready to answer some really complex questions. If you are interested in watching, but aren't able to join us live in Baltimore, you can join us virtually on our Facebook page: https://www.fa

3 Pediatric Neurology: https://kwnsfk27.r.eu-west-1.awstrack.me/L0/https:%2F%2Fauthors.elsevier.com%2Fa%2F1msSX39uenuW-p/1/0102019d45a03fba-811edca3-8989-468b-9643-485e4dbb08ec-000000/Iz_uBqmcZFBBt0i6e-m9KRsxRiY=472 Dawn B. Lammert, MD, PhD Carl E. Stafstrom, MD, PhD, Sanaya Shenoy, MSPH , Heather Riordan, MD, Jee A. Bang, MD, MPH

Porter Ranch, CA — For decades, James* was the very definition of success: a respected attorney, a devoted husband, and the proud patriarch of a thriving family. But behind the closed doors of his well-appointed home, a different story was quietly unfolding—one that would culminate in a devastating diagnosis at the age of 70. It began innocuously enough in his early 40s, with persistent, but light, movement in his leg, known as chorea. His wife, unaware of its significance, d

My cellphone has an app that shows me photos from years gone by. Memories for each day. Today's memories were a run of a lifetime with my husband, Nathan. Glimpses from dating through wedding, birth of our child, to now. When I say this, don’t take it wrong. I love my husband. But I sure miss the man I began dating, fell for, and married. I miss the walks, just being together, living life without having to always think of solutions before the problems. Most people can say si

Community Update: Novartis’ INVEST-HD Phase 3 trial: Site Initiations in USA and Canada March 16th, 2026 Dear Huntington’s Disease Community, We are delighted to share an update on the development of votoplam (formerly PTC518). Last month we shared the news that the votoplam Phase 3 clinical trial, INVEST-HD, has been posted on ClinicalTrials.gov . Further progress to initiate the study has been made since then: • We have activated our first sites for the study. This mean

s “To go back in a rare disease for sham surgeries is unethical,” Katie Jackson, president and CEO of Help 4 HD International, told BioSpace. “You’re asking people to get a sham surgery and sit in a placebo-like hold for one year, two years, three years,” preventing patients from participating in other clinical trials or from being able to receive AMT-130 in a future open-label extension study if they become too ill. Read the full article here: https://www.biospace.com/fda/un

Katie Jackson's response can be read at the following link: https://news.bloomberglaw.com/pharma-and-life-sciences/fda-push-for-10-hour-fake-brain-surgery-trial-raises-concerns

For ANY questions: medinfo@uniqure or 1-866-520-1257

FDA Seeks Feedback from Rare Disease Community The U.S. Food and Drug Administration (FDA) has opened a public docket to solicit input from the rare disease community. The FDA docket, available here , invites public comments from parties engaged in the design and conduct of rare disease clinical trials, including sponsors, investigators, patients, and patient advocates. This docket includes two distinct opportunities for feedback: The Center for Drug Evaluation and Research’s