EHDN & Enroll-HD Strasbourg 2024 | Presentations

EHDN & Enroll-HD 2024 was held September 12-14 in Strasbourg, France, an event that combined the biennial EHDN Plenary Meeting and the Enroll-HD Congress into a joint meeting. Recorded presentations from the Enroll-HD component of this meeting are displayed below.

Video Index

Keynote Lecture

Sarah Tabrizi; University College London | Huntington’s disease now – Mechanisms and therapeutics

Genetic Modifiers and Somatic Mosaicism

Bob Handsaker; Harvard Medical School | The “ticking DNA clock”: Somatic expansion of the Huntingtin CAG repeat and its biological sequelae

Davina Hensman Moss; University College London | Investigating intergenerational HTT-CAG repeat variability: Sperm-CAG

Development and Aging in HD

Oliver Bartley; Cardiff University | Epigenetic and transcriptional characterization of hPSC-MSNs

Sandrine Humbert; Paris Brain Institute | Contribution of an abnormal brain development to Huntington disease

Keynote Lecture

Harry Orr; University of Minnesota | Building bridges between CAG triplet (glutamine) repeat disorders – HD and SCA1: a comparative analysis

Practical Deployment of HD-ISS in Clinical Research

Cristina Sampaio; CHDI | HD-ISS overview: Opportunities and challenges for the future

Jeffrey Long; University of Iowa | Using the HD-ISS for planning clinical trials

Panel discussion on the HD-ISS: Walid Abi-Saab (uniQure), Peter McColgan (Roche), Amy-Lee Bredlau (PTC Therapeutics), Glenn Morrison (Annexon Biosciences)

Advances Obtained Through Enroll-HD Part 1: Biomarkers and Modelling

Katrin Barth; EHDN | Enroll-HD biosamples and data: Scope and distribution

Jong-Min Lee; Massachusetts General Hospital | How the GWAS exemplifies what you can do with Enroll-HD data: Journey to identify genetic modifiers of HD

Douglas Langbehn; University of Iowa | NfL serum data from Enroll-HD samples to explore this biomarker

James Mills; University of Iowa | Modelling using Enroll-HD data – Focus on PIN HD

Advances Obtained Through Enroll-HD Part 2: Clinical Trial and Clinical Study Support

Jenny Townhill; EHDN | Enroll-HD support for clinical trials

Marcelo Boareto; Roche | Placebo response in HD: Comparing placebo arm in GENERATION HD1 vs Enroll-HD data

Jamie Hamilton; CHDI | Quantifying indirect and out-of-pocket costs in HD (HD Charge study)

Joaquim Ferreira; University of Lisbon | Envisioning the future of clinical research with Enroll-HD 2.0

Keynote Lecture

Michael Panzara; Neurvati Neurosciences | Use of innovative trial designs to accelerate drug development in Huntington’s disease

The Next Frontier of Enroll-HD

Swati Sathe; CHDI | Enroll-HD 2.0: What is changing and why?

Matthew Roche; CHDI | selfEnroll-HD: a smartphone-based study and research platform

Advances Obtained Through Enroll-HD Part 3: Novel Clinical Trial Design

Priyantha Herath; Alnylam | Early clinical trials in HD: the need to go early and how to do it

Andrew Wood; CHDI | Early phase biomarker strategy, possibilities from imaging/PET as a target engagement biomarker

Tiago Mestre; University of Ottawa | Bridge model: Variation of seamless phase II to III trial

Patrick Weydt; University Hospital Bonn | Bridging to clinical benefit

Panel discussion between presenters

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