Huntington Disease

Huntington disease research has pivoted after tominersen with multiple new programmes in clinical development. The session covers branaplam (SMN-stabiliser repurposed for HTT lowering, suspended for safety), AMT-130 AAV gene therapy (Phase 1/2 readouts at 24-36 months), post-GENERATION HD1 lessons and trial-design pivots, NfL and mHTT biomarker development, pre-manifest care models, and emerging non-coding RNA and small-molecule therapeutics. Discussion addresses HTT-lowering antisense oligonucleotides (newer-generation after tominersen failure), pre-manifest disease and trial enrolment ethics, the role of cognitive markers in early HD, juvenile HD considerations, genetic-counselling practice changes, and the international HD registries (Enroll-HD, REGISTRY) driving research.