
Huntington’s disease research news. In plain language. Written by scientists. Editors @labscribbles @rungenado
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Skyhawk Therapeutics have received the regulatory approval needed to broaden its FALCON-HD trial to include the United States, Canada, and the UK. The expanded trial will enroll up to 400 more people with Stage 2 and early Stage 3 HD across more than 40 sites worldwide.
Roche announced it is stopping development of two of its huntingtin-lowering ASO drugs, tominersen and RG6496. This marks a disappointing end to an important chapter in HD clinical research. en.hdbuzz.net/roche-ends-two…
First participant dosed in pioneering neural stem cell trial for Huntington’s disease
A new clinical trial for HD is underway. The first participant has been dosed in REGEN4HD, a study testing whether transplanting neural stem cells is safe as a potentia… ift.tt/LTCh07A
Evolving Needs, Evolving Care: Adapting Psychological Support Across Different Stages of Huntington’s Disease
⏱️ 9 min read | Huntington’s disease affects more than movement: it also has a major impact on mental health. Researchers explore why psychologi… ift.tt/g8CA3PV
Sealing the gap: a case for slower, smarter DNA repair
⏱️ 7 min read | Your body assembles DNA like a team of workers who set and bond a pattern of tiles. A recent study shows that one special assistant might slow somatic CAG expansion and delaying HD on… ift.tt/mnjeTal
HDBuzz 2025 Annual Report: Here’s what you made possible
⏱️7 min read | In 2025, you helped us reach 329,000 people with independent, unbiased HD research news. Our 2025 Annual Report is here. Take a look at what we built together, and consider helping u… ift.tt/mle90Db
Skyhawk reports encouraging 12-month results for oral Huntington’s disease drug SKY-0515
One year into treatment, SKY-0515 continues to lower huntingtin and shows encouraging trends with signs and symptoms of HD. A larger trial is enrolling to determine … ift.tt/rendJCp
May 2026: This Month in Huntington’s Disease Research
⏱️8 min read | May 2026: 2 year data from the votoplam trial suggests disease progression may slow; AMT-130 heads to the UK for regulatory review; pigs reveal immune cells invading the HD brain; and n… ift.tt/xIgKGi8
Fixing the Recipe: Lowering a Slice of Huntingtin
⏱️7 min read | In a recent paper, scientists targeted a small, harmful piece of the huntingtin message (HTT1a) in HD mice. This reduced toxic protein clumps and delayed genetic changes, more than targetin… ift.tt/ohuZJqr
Tagging the Trash: Turnover of Toxic Huntingtin
⏱️ 5 min read | A recent study suggests that strengthening the brain's own clean-up system could slow Huntington’s disease.
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Prilenia’s Application for European Approval of Pridopidine Withdrawn
⏱️ 5 min read | Prilenia's application for European approval of pridopidine was quietly withdrawn 6 months ago, in November 2025, after the EMA had already recommended refusing the dru… ift.tt/xcZXmPe
Intruders in the Brain: What a Pig Model Reveals about Immune Cells in HD
⏱️7 min read | A recent study in pigs suggests the HD brain could be under attack from the body's own immune system. The results reveal that invading cytotoxic T cells might contri… ift.tt/elk1RxY
The Great Care Conundrum: What “Good” Care Looks Like in Huntington’s Disease
⏱️8 min read | What does “good care” look like in Huntington’s Disease? A new Dutch study asked familial and professional caregivers, and uncovered how differently it’s experie… ift.tt/3x7JjEH
A Second Path: uniQure Plans Regulatory Filing for AMT-130 in the UK
⏱️ 6 min read | While the US regulatory path for AMT-130 remains complicated, uniQure announced plans to seek approval for its HD gene therapy in the UK. Here's what that means, and wha… ift.tt/D8APmok
Two Years In: New Long-Term Extension Data from PIVOT-HD for Votoplam
⏱️10 min read | PTC Therapeutics shared 2-year data for votoplam, a daily HTT-lowering pill. Stage 2 participants showed up to 52% slowing of disease progression. Here's what the data … ift.tt/Zi1FRcO
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April 2026: This Month in Huntington’s Disease Research
⏱️ 8 min read | April 2026 showed us a toxic HTT fragment is center stage in mouse research, CRISPR is showing promise in mouse studies, AI and wearables move closer to the clinic, and 2 studies shi… ift.tt/sGtPyjH
The Number on the Scale: What the TFC Score Measures, and Why It Matters Right Now
⏱️ 10 min read | The Total Functional Capacity score has been used in Huntington’s disease research for decades. Here's what it measures, what it misses, & why it's at the… ift.tt/xT5cZ3L
Dancing With a Stranger: Understanding Apathy in Huntington’s Disease Through Caregivers
⏱️5 min read | Apathy in HD isn’t just “not caring.” It can affect relationships, routines, and emotional connection. A small study of caregivers reveals the heavy t… ift.tt/wh1dXRb
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