Gene Expression Quantification to Assess Frataxin Replacement Therapies in Friedreich’s Ataxia
Objective: To identify genetic disease and efficacy markers for Friedreich’s ataxia (FRDA) Background: FRDA is a genetic disease caused by decreased expression of the mitochondrial…Tissue Frataxin Increases After Administration of CTI-1601, a Frataxin Replacement Therapy in Development for the Treatment of Friedreich’s Ataxia
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…Validation of the Neuroprotective Potential of SLP-2 against alpha-synuclein neuropathology in a Mouse Model of Parkinson’s Disease
Objective: We aim to validate the neuroprotective potential of SLP-2 against α-syn neuropathology and neurodegeneration of dopaminergic neurons in a mouse model of Parkinson’s disease.…The mitochondrial targeted drug SBT-272 attenuates dopaminergic neuron loss, alpha-synuclein burden and neuroinflammation in a mouse model of Parkinson’s Disease
Objective: To assess the preclinical efficacy of the investigational drug SBT-272 in a mouse model of Parkinson’s Disease (PD). Background: SBT-272 is a peptidomimetic drug…Effect of Ketone Ester Supplementation on Motor and Non-Motor symptoms in Parkinson’s Disease
Objective: To investigate the effect of exogenous ketone ester supplementation on motor and non-motor symptoms in Parkinson’s Disease (PD). Background: Animal research and human pilot…Relationship of subcortical iron deposition and mitochondrial dysfunction in patients with idiopathic Parkinson’s disease
Objective: To investigate subcortical brain iron deposition as a potential predictor of the bioenergetic status in patients with idiopathic Parkinson's disease. Background: The underlying pathophysiology…Reduced cerebral energy metabolism in Parkinson’s disease in contrast to Progressive Supranuclear Palsy
Objective: To determine the significance of 31P-MRSI to differentiate between idiopathic Parkinson’s disease (PD), Progressive supranuclear palsy (PSP), and healthy control subjects. Background: Bioenergetic disturbances,…Metabolic alterations in a Drosophila model of Parkinson’s disease
Objective: Identification of metabolic alterations that could be contributing to Parkinson’s disease (PD) physiopathology and become new biomarkers or therapeutic targets for the disease. Background:…Molecular investigation of apparently healthy heterozygous Parkin mutation carriers
Objective: To investigate whether apparently healthy individuals carrying a heterozygous pathogenic Parkin (PRKN) mutation show molecular phenotypes of mitochondrial dysfunction. Background: Homozygous or compound-heterozygous mutations…Novel Recessive Mitochondrial Mutations Causing Leigh’s Syndrome and Movement Disorders
Objective: Describe discovery of novel mitochondrial mutations causing ataxia and dystonia syndromes with occurrence in childhood. Background: An African-American man at 5 years old subacutely…
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