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Programme Friday -
Thursday, September 15th, 2016

16:00-22:00   Registration Desk

Friday, September 16th, 2016
07:30-08:30  Registration Desk
Welcome and Introduction
Room: King Willem Alexander
08:30-08:40   Jean-Marc Burgunder (Bern, Switzerland)
Astri Arnesen (EHA, Norway)
08:40-09:00   EU policy on rare diseases
Jaroslaw Waligora (Rare diseases Policy Officer, European Commission)
Plenary Session I:
'Whats new in HD biology? Insights into pathogenic pathways and therapeutic targets'
Room: King Willem Alexander
Chair: Lesley Jones (Cardiff, UK)
Co-chair: Flaviano Giorgini (Leicester, UK)
09:00-09:20   RAN Translation in HD
New Starts and Directions: Understanding the Role of Hidden Expansion Proteins in HD
Laura Ranum (Florida, USA)
09:20-09:40   Somatic Expansion of Repeat Sequences in DNA
Instability of DNA Repeat Sequences: What it means when Long Repeats go on getting Longer
Darren Monckton (Glasgow, UK)
09:40-10:00   Exploring Mitochondrial Dysfunction and Dynamics in HD
Cellular Powerplants: How and Why they go Wrong in HD
Flaviano Giorgini (Leicester, UK)
10:00-10:20   An overview of Energy Metabolism in HD as a Therapeutic Target
Cellular Powerplants: How we might correct their Dysfunction in HD
Robert Pacifici (Los Angeles, USA)
10:20-10:30   Panel discussion
10:30-11:00   Tea/Coffee Break

Plenary Session II:
'Switching Huntingtin Off: Gene-Targeted Therapies'
Room: King Willem Alexander

Chair: Sarah Tabrizi (London, UK)
Co-chair: Jean-Marc Burgunder (Bern, Switzerland)
11:00-11:20  RNA Targeted Therapies, Mechanisms and the Challenges of Delivery - in HD
Douglas Macdonald (Los Angeles, USA)
11:20-11:40   RNA Targeted Therapies – the Experience from SMA and Duchennes Muscular Dystrophy
Francesco Muntoni (London, UK)
11:40-12:00  DNA Targeted Therapies – Zn Finger Approach
Matt Chiocco (Boston, USA)
12:00-12:30   Panel discussion
Poster Viewing
12:30-14:00  Poster Viewing
Room: Onyx & Amazon Foyer
12:30-14:00   Buffet Style Lunch
Parallel Session I:

Parallel - Science Session:
'Large animal models in HD'
Room: King Willem Alexander
Chair: Marta Valenza (Milan, Italy)
Co-Chair: Jenny Morton (Cambridge, UK)
14:00-14:15   Conceptual Overview: What Model to use for what Purpose?
David Howland (Princeton, USA)
14:15-14:30   Leaders not Followers: Sheep as Large Animal Models of Huntington's Disease
Jenny Morton (Cambridge, UK)
14:30-14:45   HD Minipigs - Behavioral and Imaging Protocols transitioning into Preclinical Trials
Ralf Reilmann (Münster, Germany)
14:45-15:00   Longitudinal Assessment of a Nonhuman Primate Model of Huntington's Disease
Anthony Chan (Atlanta, USA)
Parallel - Clinical Practice Session:
Room: Mississippi
Chair: Tim McLean (Edinburgh, UK)
Co-Chair: Jamie Levey (New York, USA)
14:00-14:15   How does Enroll-HD expedite/facilitate the conduct of Clinical Trials?
Tim McLean (Edinburgh, UK)
14:15-14:30   How does Enroll-HD advance Human Research in HD?
G. Bernhard Landwehrmeyer (Ulm, Germany)
14:30-14:45   How does Enroll-HD help improve Care?
Jan Frich (Oslo, Norway)
14:45-15:00   Discussion
Short Communications I:
'Scientific Presentations: 10’ – selected from submissions'

Chair: Michael Orth (Ulm, Germany)
Co-Chair: Maria Björkqvist (Lund, Sweden)
15:15-16:15   Several presentations selected from submitted abstracts
    Systemic Administration of a Novel Aav Variant Results in widespread and efficient Gene Transfer in R6/2 Mice
Pamela Farshim (University College London)
    Striatal Neuropathology induced by Hypothalamic Overexpression of Huntingtin Fragments using Adeno-associated Viral Vectors in Mice
Rana Soylu (Lund University)
    Detection of the Aberrantly Spliced Exon 1 – Intron 1 Htt Mrna in HD Patient Post Mortem Brain Tissue And Fibroblast Lines and Comparison of the Effect of a pure Cag Repeat and mixed Cagcaa Repeat on the Extent to which the Htt Gene is aberrantly spliced in Knock-In Mice
Gillian Bates and Nadira Ali (University College London)
    Cognitive Training Improves Disease Symptoms in a Mouse Model of Huntington's Disease
Emma Yhnell (Cardiff University)
    HD Clarity: A Multi-Site Cerebrospinal Fluid Collection Initiative to facilitate Therapeutic Development for Huntington’s Disease
Ed Wild (University College London)
16:15-16:45   Tea/Coffee Break
Plenary Session III:
'Keeping brain cells functioning: Targeting neural recovery and replacement'
Room: King Willem Alexander

Chair: Monica Busse (Cardiff, UK)
Co-chair: Jean-Marc Burgunder (Bern, Switzerland)

16:45-17:05   Can exercise and physical therapy alter disease progression in Huntington's Disease?
Lori Quinn (Cardiff, UK)
17:05-17:25   Exercise Induced Brain Repair in Neurodegeneration: What can we learn from Parkinson’s Disease?
Beth Fisher (Los Angeles, USA)
17:25-17:45   HD Brain-Train: neuroplasticity as a target to improve function in Huntington's Disease
Marina Papoutsi (London, UK)
17:45-18:05   Is cell replacement a feasible option for Huntington’s disease?
Anne Rosser (Cardiff, UK)
Roundup I: Lessons of the day
Room: King Willem Alexander
18:15-18:30   Lessons of the day
Ethan Signer (New York, USA) and Allan Tobin (USA)
Evening get together
19:00-19:30   Adventurer with a Cause
Lysle Turner (South Africa)
Room: Mississippi

19:00-21:00   Poster Viewing
19:30-21:00  Film: Twitch
Room: King Willem Alexander
   Film: The Inheritance
Room: Mississippi
19:30-21:00   Meet the Expert
For HD Family Members and Students

    Mental Health Problems of HD
Erik van Duijn, Alzbeta Mühlbäck, David Craufurd
Languages: Dutch, German, English
Room: Amazon

    Everyday Life as an HD Gene Carrier
Michael Orth
Languages: German, English, Spanish
Room: Yangtze 2

    Clinical Trials: What does it mean to be in a Clinical Trial
Berry Kremer, Ralf Reilmann
Languages: Dutch, German, English
Room: Yangtze 1

    Advanced Care
Anthony Lechich, Tom Stor
Languages: Dutch, English
Room: Africa

    Coping as a Caregiver and Family Member
Aad Tibben, Astri Arnesen
Languages: Dutch, English, Norwegian
Room: South America