Articles tagged "Disease-modifying strategies"

MDS Virtual Congress 2020
Translatable biomarkers in gene therapy for Huntington’s Disease: learnings from pre-clinical studies
A. Valles, M. Evers, A. Stam, M. Sogorb-Gonzales, C. Brouwers, C. Vendrell, S. Acar-Broekmans, V. Fodale, A. Bresciani, R. Nieuwland, Z. Ellederova, J. Motlik, M. de Haan, B. Leavitt, S. van Deventer, P. Konstantinova (Amsterdam, Netherlands)
Objective: We have developed AAV5-miHTT, a recombinant AAV-based gene therapy expressing an engineered microRNA that specifically binds to HTT exon1, resulting in lowering of both…
MDS Virtual Congress 2020
Exploring dopamine transporter single-photon emission computed tomography quantification as a measure of disease progression in idiopathic Parkinson’s disease
J. Gurwell, R. El Khouli, G. Quintero, J. Slevin, M. Yazell, C. van Horne (Lexington, KY, USA)
Objective: To evaluate the ability of DAT SPECT quantification to track disease progression in subjects with idiopathic Parkinson's disease (PD) by comparing baseline scans to…
MDS Virtual Congress 2020
Phase I-II first-in-human clinical trial of intraputamenal CDNF in Parkinson’s disease: Topline results of the six-month double-blind placebo-controlled main study and the six-month active treatment extension study
S. Booms, H. Huttunen, J. Koskinen, M. Sjögren, M. Saarma, M. Woolley, B. Murphy, P. Fielder, J. Baker, P. Skinner, J. Rinne, A. Varrone, M. Andreasson, G. Paul-Visse, R. Kivisaari, H. Bjartmarz, G. Lind, P. Almqvist, F. Scheperjans, H. Widner, P. Svenningsson (Espoo, Finland)
Objective: To assess the safety and tolerability and to explore the preliminary efficacy of intermittent intraputamenal monthly infusions of CDNF in subjects with advanced idiopathic…
MDS Virtual Congress 2020
Phase I-II first-in-human clinical trial of intraputamenal CDNF in Parkinson’s disease: Exploratory fluid-based biomarker endpoints of the 12-month treatment period
H. Huttunen, S. Booms, H. Abdesselem, N. Majbour, O. El-Agnaf, I. Poggiolini, L. Parkkinen, A. Cryar, E. Sirka, J. Harris, M. Dove, M. Sjögren, M. Woolley, B. Murphy, P. Fielder, J. Baker, P. Skinner, M. Andreasson, G. Paul-Visse, R. Kivisaari, H. Bjartmarz, P. Almqvist, P. Almqvist, G. Lind, F. Scheperjans, H. Widner, P. Svenningsson (Espoo, Finland)
Objective: To explore fluid-based biomarkers in subjects with advanced idiopathic Parkinson’s disease (PD) before and after intermittent intraputamenal monthly infusions of CDNF. Background: CDNF is…
MDS Virtual Congress 2020
Safety, Tolerability, and Pharmacodynamic Profile of DNL201 at dose levels demonstrating LRRK2 inhibition in Parkinson’s Disease Patients with and without LRRK2 mutations
D. Jennings, P. LeWitt, D. Kern, I. Goodman, A. Siderowf, O. Omidvar, A. Ellenbogen, J. Aldred, R. Macuica, S. Huntwork-Rodriguez, C. Ho, M. Troyer (South San Francisco, CA, USA)
Objective: To evaluate safety, tolerability, and target engagement of DNL201, a LRRK2 inhibitor, in Parkinson’s disease (PD) patients. Background: Mutations in LRRK2 impact both familial…
MDS Virtual Congress 2020
Phase I-II first-in-human clinical study of intraputamenal CDNF in Parkinson’s disease: Exploratory PET imaging endpoints of the 12-month treatment period
V. Kerstens, P. Fazio, J. Johansson, T. Granberg, S. Booms, H. Huttunen, M. Sjögren, M. Woolley, B. Murphy, P. Fielder, J. Baker, P. Skinner, M. Andreasson, G. Paul-Visse, R. Kivisaari, H. Bjartmarz, G. Lind, P. Almqvist, F. Scheperjans, H. Widner, P. Svenningsson, J. Rinne, A. Varrone (Stockholm, Sweden)
Objective: To test if intermittent intraputamenal monthly infusions of CDNF have an effect on striatal dopamine transporter (DAT) binding, as measured by PET, in subjects…
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:…
2019 International Congress
Safety of Bone marrow-derived Allogeneic Mesenchymal Stem Cells in Parkinson’s disease Patients
M. Schiess, J. Suescun, T. Ellmore, MF. Doursout, E. Furr -Stimming, Z. Mei, A. Gee (Houston, TX, USA)
Objective: Prove safety and tolerability of allogeneic mesenchymal stem cells (MSC) purified from bone marrow derived from a healthy adult and delivered intravenously in escalated…