The SUNRISE-PD Study, a clinical trial of AXO-LENTI-PD: a CNS-directed gene therapy for the treatment of Parkinson’s Disease
Objective: This presentation will provide an overview of the design and key objectives of the SUNRISE-PD study. Background: Parkinson’s disease (PD) is caused in part,…MRI, Clinical, and Neuropathological Findings after Bilateral Intra-striatal Administration of rAAV5-miHTT in Non-human Primates
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…Procedural Memory Changes of Parkinson’s Disease Patients Post Participation in Boxing
Objective: Can participation in a Rock Study Boxing program improve procedural memory in people with Parkinson’s disease (PD)? Background: Procedural memory is important for daily…A safety, tolerability and biomarker update from an ongoing open-label extension study of RG6042 in adults with early manifest Huntington’s disease
Objective: To present the safety, tolerability and biomarker effects of RG6042 (previously, IONIS-HTTRX) during an open-label extension (OLE) study in adults with early manifest Huntington’s…Experimental model of developing of Parkinson’s disease
Objective: Analyzing the reserpine model of Parkinsonian syndrome Background: There are several experimental models of Parkinson's disease: reserpine, methamphetamine, 6-hydroxydopamine (6-OHDA), 1-methyl-4-phenyl-1,2,3,6-tetrahydropyridine - substances that…Sustained mutant huntingtin lowering in the brain and cerebrospinal fluid of Huntington disease minipigs mediated by AAV5-miHTT gene therapy
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)…Systematic review of Mucuna Pruriens as a treatment for Parkinson’s disease
Objective: To conduct a systematic review of the therapeutic evidence for Mucuna Pruriens (MP) in the treatment of Parkinson’s disease (PD). Background: MP or Velvet bean is a…Enabling efficient use of digital health technologies to support Parkinson’s disease drug development through precompetitive collaboration
Objective: To describe the Critical Path for Parkinson’s (CPP) consortium approach to advance the field of digital technologies in clinical drug development for Parkinson’s disease…Increasing chances of detecting a neuroprotective signal in PhI/II PD clinical trials?
Objective: Design an early CT program to detect a neuroprotective signal in PD. Background: Attempts to develop PD modifying therapies have been largely disappointing so…A single nigral injection of human ENGRAILED-1 induces long-lasting behavior benefit in an experimental primate model of Parkinson Disease
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…
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