HDYO

@HDAwareness2024 Here’s the complete video - youtu.be/LUBAtgG8GWs?si…

See this Instagram post by @hdyofeed instagram.com/p/DVjHtQ2APmC/…

@laurencurehd @US_FDA Here’s the complete video - youtu.be/LUBAtgG8GWs?si…

A Message from @HDYOFeed The recommendation by the @US_FDA to include an imitation “sham” surgery control in future gene therapy trials for Huntington’s Disease raises significant ethical and practical concerns. While rigorous trial design is essential to demonstrate efficacy, the use of a sham neurosurgical procedure—requiring participants to undergo the risks of major surgery to the head including a lengthy time period under anesthesia without the possibility of therapeutic benefit—places an unnecessary burden on individuals already facing a progressive and fatal condition. People living with Huntington’s Disease and their families often volunteer for research with the hope of contributing to meaningful progress. While we know all trials come with some level of risk, exposing them to invasive procedures without potential benefit may undermine trust and willingness to participate in future trials. HD is a slow progressing disease presenting huge burdens for families and creating unique challenges to trial designs. Given the seriousness of the procedure and the urgent need for disease-modifying therapies, alternative trial designs that maintain scientific integrity while minimizing avoidable risk to participants should be prioritized. HDYO is committed to working alongside the Huntington’s Disease community, advocacy organizations, scientists, and regulators to promote ethical, patient-centered research approaches that accelerate the development of safe and effective therapies for people living with Huntington’s disease. We wanted to address some of the topics and questions we have been receiving this week. Those questions include a better understanding of how observational studies like Enroll-HD and HD-Clarity play a part in trials and placebos in a gene therapy. Board members, researchers and family members, Emily Leestma and Lauren Byrne join Jenna Heilman for the discussion. instagram.com/p/DVjHtQ2APmC/…

Meet Banaz, an inspiring ambassador for HDYO from the Kurdistan region in Iraq! She's bravely sharing her journey with Huntington's Disease and making a difference in the Middle East. #whatishappening #huntingtonsdisease #hope #impact

@l_e_whyte @RepAuchincloss

HDYO team reacts to the recent @US_FDA recommendations for sham surgery in gene therapy for #Huntingtonsdisease community. Full statement below ⬇️ youtu.be/LUBAtgG8GWs @uniQure_NV @laurencurehd

Together, we will continue to fight for you💙💜💙 @HDSA @HDReach @HDYOFeed @hdfcures

HDYO would like to wish all who celebrate a very Happy Easter! Have a great day!

We are committed to creating a community that is built on the foundation of making every single member feel valued and empowered. Together, we can support each other to create change 💙💛💚🩷 #HDYO

Happy Passover! The evening of Saturday 12 April marks the start of Passover which lasts until Sunday 20 April. HDYO wishes everyone who celebrates a joyous time.

🎉 Big News! 🎉 We just launched our brand-new Research Terminology catalog! 🧠✨ Dive into the latest terms and concepts in Huntington’s disease research. The definitions are designed to be understandable and can be easily translated. Check it out now! #HDYO #HuntingtonsDisease

Thank you to our sponsors! @TevaUSA , @uniQure_NV , @Roche, @neurocrine , @WaveLifeSci and the Griffin Foundation.

Watch full video: youtu.be/AiunlpqRTRs HDYO Ambassadors weigh in on the recent study looking at early progression in Huntington’s disease and how that may impact how young people view research. @EHDN_GRANTM

The Huntington’s Disease Society of India (HDSI) will host HDSI International Conference. Bringing together global experts, researchers, clinicians, patients, caregivers, policymakers. Understanding, treatment, and care of Huntington’s Disease @HDYOFeed @cbmnimhans @NIMHANS_BLR

We are excited to be featured in the latest edition of HD Insights regarding young people’s need for support first. Thank you @HuntingtonSG! huntingtonstudygroup.org/hd-insights/wh…

We would like to wish everyone celebrating this joyous occasion a wonderful Eid Mubarak! From everyone at HDYO.

Grateful for the opportunity to have a conversation about our AMT-130 program for #huntingtonsdisease @HDYOFeed’s congress in beautiful Prague. Thank you Ashley and Lauren for the great collaboration!

And that’s a wrap! It has been an amazing three days at @HDYOFeed’s congress in Prague! Thank you to Jenna, Lauren and the entire team who put this fantastic conference together. These gatherings give us the information and inspiration we need to move our AMT-130 program forward!

@ScottishHD Well done!

Fantastic news! We have been awarded £100,000 over three years from Children In Need to reach young people aged 18 and under in Highland.

British occupational therapist Alex Fisher and Venezuelan filmmaker Gindel Delgado (2024) collaborated to document the lives of two Huntington's disease caregivers in Barranquitas Venezuela over a two-week period. Today it is Huntington's appreciation day, and we send them our forever gratitude for aiding those w/ a disease that is much more than the dance-like movement disorder (chorea) we see on the outside. About their project: - Alex and Gin share a deep commitment to the HD community. Alex works in a Huntington’s disease service in the UK, while Gindel, an Emmy-nominated filmmaker, has been documenting this community since his work on the film "Dancing at the Vatican." - March 23rd honors and expresses appreciation for the extraordinary individuals living with HD and their cooperation w/ the scientific community. - 'Gratitude Day' particularly acknowledges the people of Zulia in Venezuela, where scientists originally mapped the disease, leading to substantial advancements in understanding HD. - Given the current geopolitical situation that hinders on-the-ground research, the efforts of the humanitarian organization Factor H have become increasingly vital. - The local community, initially wary of outsiders, now trusts Alex and Gin, bestowing upon them the surname Soto, reflective of the town. This trust enabled them to capture the unique experiences of those living with HD in Barranquitas, as well as the resilience of individuals such as Jesus and Maribella. - Jesus, a teacher with a steady but modest income, supports his family, which once included four relatives with HD. Maribella, with 14 members in her household, earns a living as a caregiver through the Factor H caregivers program, which Alex designed in 2024 and later received the Amgen Prize for its innovative approach. - The caregivers program provides financial stability and support to individuals like Maribella and facilitates the care of community members like Celina. - The program has enabled Factor H to begin to establish a 'multi-functional community hub.' - This facility will offer essential services such as food, showers, and potentially palliative care for homeless individuals with HD, and ever the optimists it may also serve as a future site for research activities. My take: BRAVO! I went to Maracaibo and Barranquitas VZ on Nancy Wexler's NIH trip ~25 years ago as a rating neurologist for early research studies. Alex and Gin are heroes and you should reach out if interested in the caregivers project and in their documentary. The film is 27-minutes and FREE to HD educational and advocacy organizations. While we search for better treatments, and perhaps a cure remember – bis vivit qui bene vivit – he lives twice who lives well. Let's appreciate these families and caregivers and partners and help them to live well. youtube.com/watch?v=EPLjSr… factor-h.org/alex-fisher-re… @HDSA #huntingtonsdisease @alex_fisher1969 @FixelInstitute