MDS Abstracts

Abstracts from the International Congress of Parkinson’s and Movement Disorders.

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Articles tagged "Disease-modifying strategies"

  • 2019 International Congress

    Translating Preclinical Data to a Human Equivalent Dose for AMT-130 AAV Gene Therapy for Early Manifest Huntington’s Disease

    M. Evers, M. de Haan, A. Valles-Sanchez, E. Sawyer, S. Gill, R. Roos, S. van Deventer, P. Konstantinova, J. Higgins (Amsterdam, Netherlands)

    Objective: To extrapolate the human equivalent dose (HED) for a Phase I/II clinical trial in adults to explore the safety, tolerability, and efficacy of striatally-administered…
  • 2019 International Congress

    MRI, Clinical, and Neuropathological Findings after Bilateral Intra-striatal Administration of rAAV5-miHTT in Non-human Primates

    J. Higgins, B. Blits, L. Spronck, A. Valles-Sanchez, M. Evers, S. van Deventer, P. Konstantinova, M. de Haan (Lexington, MA, USA)

    Objective: To assess the effects of intra-striatal administration of a huntingtin protein (HTT) lowering gene therapy in macaca fascicularis non-human primates (NHP). Background: The aggregation…
  • 2019 International Congress

    Magnetic Resonance Spectroscopy Evaluation of Neuronal Integrity and Astrocytosis in a Phase 2 Study of Laquinimod as a Treatment for Huntington Disease (LEGATO-HD)

    B. Leavitt, R. Reilmann, M. Gordon, K. Anderson, A. Feigin, S. Tabrizi, J. Stout, P. Piccini, B. Russell-Schulz, A. Mackay, B. Borowsky, G. Rynkowski, R. Volkinshtein, J. Savola, M. Hayden (Vancouver, BC, Canada)

    Objective: Evaluate the effect of laquinimod on putaminal and frontal white matter markers of neuronal integrity and astrocytosis using magnetic resonance spectroscopy (MRS) in patients…
  • 2019 International Congress

    Brain MRI Volume Changes after 12 months laquinimod treatment of Huntington disease (LEGATO-HD)

    R. Reilmann, M. Gordon, A. Feigin, K. Anderson, S. Tabrizi, B. Leavitt, J. Stout, P. Piccini, N. Hobbs, R. Manber, B. Borowsky, G. Rynkowski, R. Volkinshtein, J. Savola, M. Hayden (Victoria, Australia)

    Objective: To explore the effect of laquinimod on brain volume in patients with Huntington Disease (HD). Background: Volume loss in caudate and other brain regions…
  • 2019 International Congress

    Quantitative Motor (Q-Motor) Assessments Suggest a Beneficial Central Effect of Laquinimod in a Phase II Study in Huntington Disease (LEGATO-HD)

    R. Reilmann, M. Gordon, R. Schubert, K. Anderson, A. Feigin, S. Tabrizi, B. Leavitt, J. Stout, P. Piccini, B. Borowsky, G. Rynkowski, R. Volkinshtein, J. Savola, M. Hayden (Muenster, Germany)

    Objective: Evaluate the change from baseline at week 52 in Q-Motor (Quantitative Motor) measures, exploratory, standardized, and rater-independent outcomes in the LEGATO-HD study. Background: LEGATO-HD…
  • 2019 International Congress

    Sustained mutant huntingtin lowering in the brain and cerebrospinal fluid of Huntington disease minipigs mediated by AAV5-miHTT gene therapy

    A. Vallès-Sanchez, A. Stam, C. Brouwers, J. Klíma, B. Bohuslavová, R. Pintauro, M. Sogorb-Gonzalez, L. Paerels, V. Fodale, A. Bresciani, Z. Ellederová, B. Blits, J. Motlik, S. van Deventer, M. Evers, P. Konstantinova (Amsterdam, Netherlands)

    Objective: To assess the translatability and long-term efficacy of gene therapy-mediated huntingtin (HTT)-lowering in a large animal model of Huntington disease (HD), transgenic HD (tgHD)…
  • 2019 International Congress

    Path to Prevention (P2P) – Developing a Prodromal PD Progression Biomarker Program

    K. Marek, A. Siderowf, C. Coffey, T. Foroud, V. Arnedo, E. Flagg, S. Chowdhury, K. Kieburtz, L. Chahine, J. Seibyl (New Haven, CT, USA)

    Objective: The overall objective of the Path to Prevention (P2P) initiative is to identify clinical, imaging, and biologic markers of progression during the prodromal phase…
  • 2019 International Congress

    A single nigral injection of human ENGRAILED-1 induces long-lasting behavior benefit in an experimental primate model of Parkinson Disease

    A. Prochiantz, E. Pioli, C. Friedel, K. Moya, E. Bézard, A. Bousseau (Paris, France)

    Objective: Evaluate the efficacy of a single nigral injection of human ENGRAILED-1 (hEN1) in a recognized chronic MPTP macaque model. Background: EN1 homeoprotein has been…
  • 2019 International Congress

    Substantia Nigra (SN) Hyperechogenicity as seen in Transcranial Sonography (TCS) is a reliable disease progression marker from Stage I to Stage II in Early Onset Parkinson’s Disease (EOPD)

    S. Ravi, V. Shivkumar, D. Dang, T. Gilmour, A. Espay, J. Wang, T. Subramanian, K. Venkiteswaran (Hershey, PA, USA)

    Objective: To test the hypothesis that SN hyperechogenicity seen in transcranial sonography (TCS) can be used as a reliable disease progression marker from stage I…
  • 2019 International Congress

    Evaluation of neuroprotection by human ENGRAILED-1 in a chronic AAV-hα-synuclein-A53T (AAV-A53T) rat model of Parkinson Disease

    A. Prochiantz, G. Porras, A. Simonnet, E. Pioli, C. Friedel, E. Bézard, A. Bousseau (Paris, France)

    Objective: Investigate the long-term neuroprotective effect  of a single bilateral injection of human ENGRAILED1 (hEN1) in a rat α-synuclein model of Parkinson Disease (PD). Background:…
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