Benvenuto su European Huntington's Disease Network
La nostra Missione
L’EHDN è un network indipendente e non profit dedicato alla promozione della ricerca, alla conduzione di trial clinici e al miglioramento delle cure destinate alle persone affette dalla Malattia di Huntington. Il network comprende clinici, ricercatori e persone affette dalla MH, insieme per realizzare la nostra missione. PER SAPERNE DI PIÙ SULL’EHDN
Find out about voting for the Executive Committee, Scientific and Bioethics Committee, and the fifth constitutional amendment here.
EHDN Platform Meeting: Tuesday, 29 September 2026
Tominersen: GENERATION HD2 Results and Key Learnings
Following Roche’s community letter announcing the discontinuation of GENERATION HD2, and EHDN’s Executive Committee statement, we will welcome Peter McColgan, Global Development Leader at Roche, to present the results and key learnings from the Phase 2 clinical trial testing tominersen in Huntington’s disease.
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On Thursday 9 July 2026, Roche announced the discontinuation of two EHDN-endorsed studies:
• GENERATION HD2 (clinicaltrials.gov/study/NCT05686551): A phase 2/open-label extension study of tominersen compared with placebo in Huntington’s disease (HD).
• POINT-HD (clinicaltrials.gov/study/NCT07246941): A phase 1 study of RG6496 delivered intrathecally compared with placebo in HD.
The community letter from Roche can be read in full here.
On 30 June 2026, Skyhawk Therapeutics shared further 12-month data from the ongoing phase 1/2 trial of SKY-0515 (also being tested in the EHDN-endorsed phase 2/3 study). Utilising a propensity score-weighted comparison – using Enroll-HD and TRACK-HD natural history datasets – favourable and consistent trends for participants receiving SKY-0515 were found across the four cUHDRS subcomponents. Skyhawk also shared 12-month data from the Clinician and Patient Global Impression of Change surveys, indicating an absence of clinician- and patient-assessed disease progression.
Click here for details of all ongoing and completed EHDN-endorsed studies.
EHDN/MDS (ES) Fellowship programme - applications for 2027 programme opening on 1st September 2026
The main objective of the fellowship programme is to motivate and facilitate the training of early career professionals (neurologists, psychiatrists, psychologists, physiotherapists, clinical geneticists or other healthcare professionals) working in HD clinical practice through the sharing of knowledge and experience. Fellows selected for the programme spend six weeks working in a highly regarded, multi-disciplinary HD clinic in Europe. Priority will be given to applicants from regions currently underserved in HD care and provision. Applications should clearly justify the need, either in relation to overall HD care or to specific aspects of HD care.
An assistance budget will be provided to help with the costs of the fellowship programme.MORE Deadline: Monday, 16 November at 12:00 CET
The Lesley Jones Seed Fund was designed to help researchers fast-track the pilot studies needed to apply for larger funding elsewhere. Following the 100th award, as announced in the July issue of EHDN News, we are launching the Seed Fund Impact Survey to better understand the programme’s reach and impact since inception.
Past and current recipients are invited to share their input and insights so we can identify the most successful aspects of the programme, as well as areas that could be further refined and enhanced to better support the HD community. Seed fund projects that have generated significant impact will be featured as case studies in future issues of EHDN News and on our website.
The seventh release of the Enroll-HD periodic dataset, PDS7, has been fully curated and is now available.
PDS7 contains data from 30,511 Enroll-HD study participants encompassing 112,992 study visits, making it one of the largest cohort datasets available to researchers. Read more here.
Active EHDN endorsed* studies
*EHDN endorsement: The EHDN endorses protocols it finds to be of a high scientific and ethical quality. ...
• Alnylam Pharmaceuticals – ALN-HTT02-001
• Cardiff University DEVISE-HD
• Novartis Pharmaceuticals – INVEST-HD
• Skyhawk Therapeutics – FALCON-HD
• UniQure Biopharma B.V. – HD GeneTRX2
• Vico Therapeutics – VO659-CT01
• UniQure Biopharma B.V. – HD GeneTRX2
Alnylam Pharmaceuticals Global study endorsed* by EHDN
Study aims and goal: ALN-HTT02 is a novel C16-siRNA conjugate for huntingtin-lowering. This Phase Ib study aims to evaluate ALN-HTT02 in adults with Huntington’s Disease. No. of study participants and sites: The study plans to enrol participants from 2 sites in Australia and New Zealand. For further information see the clinical trial registration.
*EHDN endorsement: The EHDN endorses protocols it finds to be of a high scientific and ethical quality. The endorsement decision is made on the basis of protocol review and discussion by the EHDN Executive Committee following recommendations from the independent EHDN Scientific and Bioethical Advisory Committee. Trial protocol endorsement should not be viewed as sponsor promotion or recruitment incitement. [EHDN endorsed studies overview]
Cardiff University DEVISE-HD study endorsed* by EHDN
Study aims and goal: Double-blind randomised controlled trial of sertraline, a selective serotonin reuptake inhibitor, to assess the feasibility of conducting a full-scale RCT of the efficacy of antidepressants in HD. The study will also assess some of the current clinical and methodological uncertainties around the effect of antidepressants on HD disease progression. No. of study participants and sites: 40 participants in the UK reporting mild or moderate symptoms of depression. For further information see here.
*EHDN endorsement: The EHDN endorses protocols it finds to be of a high scientific and ethical quality. The endorsement decision is made on the basis of protocol review and discussion by the EHDN Executive Committee following recommendations from the independent EHDN Scientific and Bioethical Advisory Committee. Trial protocol endorsement should not be viewed as sponsor promotion or recruitment incitement. [EHDN endorsed studies overview]
Novartis Pharmaceuticals INVEST-HD study endorsed* by EHDN
Study aims and goal: Phase III study to investigate the efficacy, safety and tolerability of Votoplam in participants with Huntington’s Disease. No. of study participants and sites: It is planned to enrol 770 participants globally. The number of sites and countries is still to be determined. For further information see the clinical trial registration.
*EHDN endorsement: The EHDN endorses protocols it finds to be of a high scientific and ethical quality. The endorsement decision is made on the basis of protocol review and discussion by the EHDN Executive Committee following recommendations from the independent EHDN Scientific and Bioethical Advisory Committee. Trial protocol endorsement should not be viewed as sponsor promotion or recruitment incitement. [EHDN endorsed studies overview]
FALCON-HD, sponsored by Skyhawk Therapeutics, Inc., endorsed* by EHDN
Study overview: Phase 2/3 randomised, double-blind, placebo-controlled clinical trial to evaluate the safety, pharmacodynamics and efficacy of SKY-0515 in HD. Recruitment: 400 participants will be recruited across Argentina, Brazil, Canada, Chile, Colombia, Georgia, Germany, Mexico, Peru, Poland, Spain, and the UK. For further information see the clinical trial registration and www.falcon-hd.com/study-information-worldwide.
*EHDN endorsement: The EHDN endorses protocols it finds to be of a high scientific and ethical quality. The endorsement decision is made on the basis of protocol review and discussion by the EHDN Executive Committee following recommendations from the independent EHDN Scientific and Bioethical Advisory Committee. Trial protocol endorsement should not be viewed as sponsor promotion or recruitment incitement. [EHDN endorsed studies overview]
Vico Therapeutics B. V. study endorsed* by EHDN
Study aims and goal: The goal of this first-in-human clinical trial is to assess the safety and tolerability of four doses of a new study drug called VO659 in people with genetic disorders called spinocerebellar ataxia type 1, type 3 or Huntington’s disease. No. of study participants and sites: The study plans to enrol participants from 14 sites in Denmark, France, Germany, Israel, Netherlands and United Kingdom. For further information see the clinical trial registration.
*EHDN endorsement: The EHDN endorses protocols it finds to be of a high scientific and ethical quality. The endorsement decision is made on the basis of protocol review and discussion by the EHDN Executive Committee following recommendations from the independent EHDN Scientific and Bioethical Advisory Committee. Trial protocol endorsement should not be viewed as sponsor promotion or recruitment incitement. [EHDN endorsed studies overview]
uniQure Biopharma EU gene therapy study endorsed* by EHDN
Study aims and goal: Phase Ib/II Study to explore Safety, Tolerability, and Efficacy Signals of Multiple Ascending Doses of Striatally-Administered rAAV5-miHTT Total Huntingtin Gene (HTT) Lowering Therapy (AMT-130) in Early Manifest Huntington Disease. No. of study participants and sites: The study aims to recruit 15 participants at several sites in the US and Europe. For further information see the clinical trial registration.
*EHDN endorsement: The EHDN endorses protocols it finds to be of a high scientific and ethical quality. The endorsement decision is made on the basis of protocol review and discussion by the EHDN Executive Committee following recommendations from the independent EHDN Scientific and Bioethical Advisory Committee. Trial protocol endorsement should not be viewed as sponsor promotion or recruitment incitement. [EHDN endorsed studies overview]
I ricercatori, i clinici e le famiglie con la MH lavorano insieme all’interno dell’EHDN per realizzare la nostra missione, e chiunque sia interessato alla MH può diventare un membro EHDN. Sono, inoltre, disponibili ulteriori informazioni sulla MH, sui siti clinici e sulla partecipazione a opportunità di ricerca più vicine a te, e su come rimanere in contatto con ulteriori risorse per te e la tua famiglia.
L’EHDN supporta la ricerca e il miglioramento della clinica per tutti gli stadi della MH, dalle basi e scienze applicate attraverso lo sviluppo di nuovi trattamenti, strumenti di valutazione clinica e linee guida per le migliori pratiche cliniche, alla valutazione di fattibilità dei siti, selezione dei siti e reclutamento di pazienti per gli studi clinici.
La Malattia di Huntington (MH) è una rara malattia genetica neurodegenerativa che è causata da una mutazione di un singolo gene; ciascun figlio di un genitore affetto dalla MH ha un rischio del 50% di ereditare il gene mutato e di sviluppare prima o poi la malattia. I sintomi includono disturbi motori, comportamentali e cognitivi, che insorgono solitamente in età adulta.
Ci sono terapie che possono alleviare alcuni dei sintomi legati alla MH e migliorare la qualità di vita dei pazienti con MH e delle loro famiglie. I gruppi di lavoro dell’EHDN hanno sviluppato le linee guida per le migliori pratiche cliniche per la MH, e il Comitato per il Miglioramento delle Cure, parte di Enroll-HD, sta raccogliendo informazioni sulle pratiche cliniche per la MH in tutto il mondo.
