MDS Abstracts

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

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Articles tagged "Disease-modifying strategies"

  • 2022 International Congress

    Designing a multi-arm multi-stage trial for Parkinson’s

    M-L. Zeissler, M. Burnell, T. Barber, Y. Ben-Shlomo, R. Chapman, C. Clarke, C. Counsell, M. Edwards, R. Ellis-Doyle, C. Gonzales-Robles, A. Jewell, G. Mills, J. Rudiger, D. Salathiel, S. Whipps, A. Whone, T. Foltynie, C. Carroll, R. Barker, J. Carpenter (Plymouth, United Kingdom)

    Objective: The Edmond J Safra ACT-PD initiative (EJS ACT-PD) is developing the first multi-arm multi-stage (MAMS) platform trial for potential disease modifying approaches for Parkinson’s…
  • 2022 International Congress

    Funding and Sustainability Challenges in Multi-Arm Multi-Stage Clinical Trials in Parkinson Disease

    C. Gonzalez-Robles, J. Ahmed, C. Bale, R. Chapman, S. Clegg, E. Deeson, R. Ellis-Doyle, V. Greaves, J. Handley, E. Henderson, K. Matthews, S. Medd-Phillips, L. Miller, G. Mills, J. Rudiger, ML. Zeissler, C. Carroll, T. Foltynie, S. Gandhi, J. Duffen (London, United Kingdom)

    Objective: The Edmond J Safra Accelerating Clinical Trials in Parkinson Disease (PD) initiative (EJS ACT-PD) aims to accelerate the identification of disease modifying treatment for…
  • 2022 International Congress

    Longitudinal Exercise is Associated with Reduced Hospitalization Risk in Parkinson’s Disease

    G. Kannarkat, M. Rafferty, S. Luo, H. Liu, K. Mills (Baltimore, USA)

    Objective: To determine if longitudinal exercise and physical therapy (PT) use is associated with reduced risk of hospital encounter in individuals with Parkinson’s disease (iwPD).…
  • 2022 International Congress

    Improving the design of clinical trials in early-stage Parkinson’s disease by using the estimand framework

    J. Anzures-Cabrera, A. Monnet, G. Pagano (Welwyn Garden City, United Kingdom)

    Objective: To show how the estimand framework can be used to improve design of clinical trials testing treatments in early-stage Parkinson’s disease (PD). Background: The…
  • 2022 International Congress

    IkT-148009 as a potential disease-modifying therapy in PD

    M. Werner, S. Karuppagounder, R. Rush, T. Kelly, T. Dawson, V. Dawson (Atlanta, USA)

    Objective: Modeling Parkinson’s disease in mice suggests c-Abl activation is required for PD initiation and progression, and therefore inhibition of c-Abl could be a strategy…
  • 2022 International Congress

    Addressing pathological alpha-synuclein aggregation using a small molecule strategy

    E. Tsika, N. Ait-Bouziad, N. Dreyfus, C. Vallet, L. Maliqi, L. Aeschbach, S. Pautet, S. Menant, I. Borovko, L. Rey-Bellet, A. Ouared, R. Migliorini, K. Piorkowska, V. Darmency, H. Kroth, S. Poli, A. Pfeifer, M. Kosco-Vilbois (Lausanne, Switzerland)

    Objective: The discovery of cell-permeable, orally bioavailable and brain penetrant small molecules with the potential of inhibiting aggregation and intracellular accumulation of pathological alpha-synuclein (a-syn)…
  • 2022 International Congress

    Fingolimod-responsive complex hyperkinetic movement disorder in neuromyelitis optica spectrum disorder

    RE. Cortes, JA. Vatanagul, G. Saranza (Cebu, Philippines)

    Objective: Highlight a complex hyperkinetic movement disorder in Neuromyelitis optica spectrum disorder exacerbation with remission on fingolimod therapy. Background: Neuromyelitis optica spectrum disorder (NMOSD) can…
  • 2022 International Congress

    Treatment Selection in Multi-Arm Multi-Stage Clinical Trials in Parkinson Disease: The Search for the Ideal Neuroprotective Drug

    C. Gonzalez-Robles, D. Byrom, R. Chapman, D. Dexter, S. Duty, R. Ellis-Doyle, E. Jabbari, G. Mills, H. Mortiboys, J. Rudiger, E. Sammler, P. Scurfield, S. Stott, G. Tofaris, L. Wei, A. Wong, ML. Zeissler, C. Carroll, T. Foltynie, O. Bandmann, A. Schapira (London, United Kingdom)

    Objective: The Edmond J Safra Accelerating Clinical Trials in Parkinson disease (PD) initiative (EJS ACT-PD) aims to accelerate the identification of disease modifying treatments for…
  • 2022 International Congress

    Selection of clinical doses for SBT101, an AAV9-hABCD1 vector for the treatment of adrenomyeloneuropathy

    D. Anderson, C. Maguire, C. Ng, Y. Gong, F. Eichler, S. Fourcase, C. Guilera, A. Pujol, A. Onieva, M. Leal-Julià, S. Verdés, A. Bosch, I. Dijkstra, S. Kemp, H. Park, T. Del Rio, T. Lutz, V. Vasireddy, S. Clark, K. Kozarsky (Bala Cynwyd, USA)

    Objective: To inform selection of doses of SBT101 for a first-in-human clinical study. SBT101 is an adeno-associated virus serotype 9 (AAV9)-based gene therapy candidate intended…
  • 2022 International Congress

    Challenges in Outcome Measure Selection in Multi-Arm Multi-Stage Clinical Trials in Parkinson Disease

    C. Gonzalez-Robles, M. Bartlett, M. Burnell, R. Chapman, R. Chaudhuri, C. Clarke, R. Ellis-Doyle, M. Hu, B. Huxford, A. Jha, M. Lawton, G. Mills, A. Noyce, P. Piccini, L. Rochester, J. Rudiger, C. Siu, D. van Wamelen, C. Williams-Gray, ML. Zeissler, H. Zetterberg, C. Carroll, T. Foltynie, R. Weil, A. Schrag (London, United Kingdom)

    Objective: The Edmond J Safra Accelerating Clinical Trials in Parkinson Disease (PD) initiative (EJS ACT-PD) aims to accelerate the identification of disease modifying treatment for…
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