MDS Abstracts

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

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Articles tagged "Experimental therapeutics"

  • 2023 International Congress

    LRRK2 PROTAC® degrader molecules induce robust biomarker responses in preclinical in vivo pharmacology studies following acute and chronic oral dosing

    J. Meredith Jr, K. Kelly, V. Guss, A. Desantis, L. Kimmel, S. Soletsky, J. Pizzano, G. Cadelina, J. Chavez, S. Sparks, A. Cacace (New Haven, USA)

    Objective: Report on the non-clinical in vivo pharmacokinetic and pharmacodynamic biomarker response to PROteolysis TArgeting Chimera (PROTAC®) molecules designed to induce degradation of leucine rich…
  • 2023 International Congress

    Development of potent, orally bioavailable, and highly selective LRRK2 PROTAC® degrader molecules as potential disease modifying therapeutics for Parkinson’s disease

    K. Kelly, J. Meredith, L. Kimmel, S. Sparks, A. Desantis, D. Bryce, R. Wilson, J. Corradi, S. Keenan, G. Cadelina, J. Pizzano, A. Cacace (New Haven, USA)

    Objective: Report on the pharmacological characteristics of small molecule PROteolysis TArgeting Chimera (PROTAC) molecules designed to induce degradation of leucine rich repeat kinase 2 (LRRK2)…
  • 2023 International Congress

    Consanguinity is the key player in complexity of Mendelian form of neurodegeneration with brain iron accumulation in Pakistan

    SHB. Rehman (Bannu, Pakistan)

    Objective: In the current work, we recognized big inbreed Pakistani family with autosomal recessive NBIA containing several affected individuals. Aim of the research work was…
  • 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…
  • 2022 International Congress

    Impact of rs6265 in the Host and Donor on Dopamine Neuron Graft Function.

    C. Szarowicz, M. Caulfield, J. Stancati, M. Vanderwerp, C. Sortwell, J. Patterson, M. Benskey, T. Collier, K. Steece-Collier (Grand Rapids, USA)

    Objective: Extend investigations into the effect of the rs6265 single nucleotide polymorphism (SNP) on dopamine (DA) neuron graft efficacy using a parkinsonian rat model. Background:…
  • 2022 International Congress

    Is there a role for safinamide in atypical parkinsonian syndromes?

    A. Silva, M. Rodrigues (Braga, Portugal)

    Objective: To search for possible benefits of safinamide in atypical parkinsonism. Background: Safinamide is a selective reversible monoamine oxidase-B inhibitor with both dopaminergic and glutamatergic…
  • 2022 International Congress

    Neurophysiological and Clinical Effects of Low-intensity Transcranial Ultrasound of the Motor Cortex in Parkinson’s Disease

    T. Grippe, Y. Shamli Oghli, G. Darmani, T. Arora, C. Sarica, C. Rinchon, J. Nankoo, C. Gunraj, R. Chen (Toronto, Canada)

    Objective: Evaluate the neurophysiological and clinical effects of transcranial ultrasound stimulation (TUS) of motor cortex (M1) in PD patients. Background: Low-intensity TUS is a novel,…
  • 2022 International Congress

    Longitudinal Program to Prevent PD (LoPP-PD): Multimodal Risk Stratification Informs Personalized Intervention

    K. Niotis, K. Akiyoshi, R. Isaacson, S. Isaacson (New York, USA)

    Objective: Longitudinal evaluation of personalized intervention strategies informed by detailed clinical and laboratory biomarker analysis to reduce relative risk of Parkinson’s disease in first degree…
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