MDS Abstracts

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

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Articles tagged "Experimental therapeutics"

  • 2022 International Congress

    Use of alternative healing for Parkinson’s disease in Kenya

    N. Fothergill-Misbah, R. Walker (Southampton, United Kingdom)

    Objective: To explore the experience of alternative healing practices by people with Parkinson’s disease in Kenya. Background: As a neurodegenerative condition, Parkinson’s disease (PD) requires…
  • 2022 International Congress

    Tissue Frataxin Increases After Administration of CTI-1601, a Frataxin Replacement Therapy in Development for the Treatment of Friedreich’s Ataxia

    D. Bettoun, T. Galas, D. Schecter, N. Ruiz, R. Clayton, J. Farmer (Bala Cynwyd, USA)

    Objective: To evaluate the pharmacodynamic (PD) and safety profiles of subcutaneous (SC) administration of multiple ascending doses of CTI-1601 in patients age ≥18 years with…
  • 2022 International Congress

    Phase 2 study evaluating safety, PK/PD, biomarkers, and efficacy of ANX005 in patients with Huntington’s disease (HD)

    R. Kumar, D. Claassen, A. Mongan, A. Grover, B. Hoehn, P. Lin, R. Arnold, E. Cahir-Mcfarland, T. Yednock, S. Keswani (Englewood, USA)

    Objective: To assess the safety, PK/PD, exploratory biomarkers, and efficacy of ANX005 in patients with, or at risk for, manifest HD. Background: Increased complement activation…
  • 2022 International Congress

    Exosomes from human dental pulp stem cells as a cell-free drug delivery vehicle in vitro and in vivo chronic PD rat model through the intranasal route targeting efficacy & bio-distribution.

    I. Datta, A. Kaushal, K. Mondal (Bangalore, India)

    Objective: To evaluate the drug encasement in human dental pulp stem cell derived exosomes and its delivery in vitro in single cell level using fluorescence…
  • 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

    An AAV9 encoding human ABCD1 (SBT101) shows functional improvement following spinal cord delivery in a rodent model of adrenomyeloneuropathy

    A. Pujol, A. Bosch, I. Dijkstra, S. Kemp, Y. Jaspers, S. Verdés, S. Fourcade, C. Guilera, M. Leal-Julià, A. Onieva, V. Vasireddy, SW. Clark, D. Anderson, K. Kozarsky (Barcelona, Spain)

    Objective: SBT101 is an adeno-associated virus serotype 9 (AAV9)-based gene therapy candidate delivering a functional copy of the human ABCD1 gene that is in development…
  • 2022 International Congress

    ADS024 attenuates striatal dopamine loss and restores movement in animal models of Parkinson’s disease

    L. Chesnel, S. Acton (Concord, USA)

    Objective: To evaluate the effect of ADS024, an oral single strain live biotherapeutic product (SS-LBP), on the pathophysiology of MPTP-induced Parkinson’s Disease (PD) phenotypes in…
  • MDS Virtual Congress 2021

    Long-term safety and activity of ecopipam (ECO), a selective D1 antagonist during treatment of pediatric patients with Tourette Syndrome

    R. Chipkin, A. Mahableshwarkar, D. Gilbert (Chicago, USA)

    Objective: To evaluate the long-term safety and activity of ecopipam (ECO) in the treatment of children and adolescents with Tourette syndrome (TS). Background: D2 antagonists…
  • MDS Virtual Congress 2021

    Homeostatic Improvement of Brain Bioenergetic Metabolism in Parkinson’s Disease: Results From A Phase 2 REPAIR-PD Clinical Trial With CNM-Au8

    R. Glanzman, J. Ren, Phd, R. Dewey, Iii, A. Rynders, K. Ho, Phd, R. Dewey, JR. (Holladay, USA)

    Objective: To determine the effects of the nanocatalytic drug, CNM-Au8, on brain energy metabolites in Parkinson’s Disease. Background: Converging lines of evidence are identifying brain…
  • MDS Virtual Congress 2021

    Phase 1/2 Open-label Dose Evaluation Study of AXO-Lenti-PD Gene Therapy for Parkinson’s Disease: Efficacy, Safety, and Tolerability Data from the Second Cohort at 6 Months

    G. Corcoran, A. Karunakara, B. Vaughn, E. Brand-Schieber, T. Foltynie, R. Barker, S. Palfi (New York, USA)

    Objective: Results from cohort 2 in the Phase 1/2 SUNRISE-PD study (NCT03720418) at 6 months (6M) post administration of AXO-Lenti-PD (ALPD) gene therapy are presented.…
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