TFEB-driven expression prevents neurodegeneration in a multiple system atrophy mouse model
Objective: To explore the potential neuroprotective effects of overexpressing the transcription factor EB in a MSA mouse model . Background: Synucleinopathies are neurodegenerative diseases characterized…Longitudinal analysis of the modified Hoehn and Yahr disease stage in PD-1101, a Phase 1b clinical study of VY-AADC01
Objective: To describe changes over the first year in disease severity and activities of daily living using modified Hoehn and Yahr (mH&Y) stage and UPDRS…Investigation of the protective efficacy of alpha-synuclein-specific antibodies in a novel neuronal cell model for synucleinopathies
Objective: To investigate the protective efficacy of different alpha-synuclein (α-Syn)-targeting antibodies in a new model of α-Syn cell-to-cell spread. Background: The 14 kDa protein α-Syn…Translating Preclinical Data to a Human Equivalent Dose for AMT-130 AAV Gene Therapy for Early Manifest Huntington’s Disease
Objective: To extrapolate the human equivalent dose (HED) for a Phase I/II clinical trial in adults to explore the safety, tolerability, and efficacy of striatally-administered…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)…
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