Exosomes from human dental pulp stem cells as a cell-free drug delivery vehicle in vitro and in vivo chronic PD rat model through the intranasal route targeting efficacy & bio-distribution.
Objective: To evaluate the drug encasement in human dental pulp stem cell derived exosomes and its delivery in vitro in single cell level using fluorescence…Selection of clinical doses for SBT101, an AAV9-hABCD1 vector for the treatment of adrenomyeloneuropathy
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…An AAV9 encoding human ABCD1 (SBT101) shows functional improvement following spinal cord delivery in a rodent model of adrenomyeloneuropathy
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…ADS024 attenuates striatal dopamine loss and restores movement in animal models of Parkinson’s disease
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…Long-term safety and activity of ecopipam (ECO), a selective D1 antagonist during treatment of pediatric patients with Tourette Syndrome
Objective: To evaluate the long-term safety and activity of ecopipam (ECO) in the treatment of children and adolescents with Tourette syndrome (TS). Background: D2 antagonists…Homeostatic Improvement of Brain Bioenergetic Metabolism in Parkinson’s Disease: Results From A Phase 2 REPAIR-PD Clinical Trial With CNM-Au8
Objective: To determine the effects of the nanocatalytic drug, CNM-Au8, on brain energy metabolites in Parkinson’s Disease. Background: Converging lines of evidence are identifying brain…Phase 1/2 Open-label Dose Evaluation Study of AXO-Lenti-PD Gene Therapy for Parkinson’s Disease: Efficacy, Safety, and Tolerability Data from the Second Cohort at 6 Months
Objective: Results from cohort 2 in the Phase 1/2 SUNRISE-PD study (NCT03720418) at 6 months (6M) post administration of AXO-Lenti-PD (ALPD) gene therapy are presented.…Overcoming clinical research challenges during a pandemic: selection and implementation of digital phenotyping solutions in a natural history study of G2019S LRRK2 Parkinson’s disease.
Objective: To identify candidate wearable devices or digital solutions suitable for conducting the Timed Up and Go (TUG) test and evaluate real-world performance remotely during…PHYSICAL ACTIVITY AT HOME IS ABLE TO REDUCE THE WORSENING IN QUALITY OF LIFE IN PARKINSON’S DISEASE DURING COVID-19 PANDEMIC
Objective: To compare the effects of physical activity (PA) at home with and without remote supervision on the motor and non-motor aspects of daily life…The PD-1102 trial in advanced Parkinson’s disease: safety and clinical outcomes from a 3-year phase 1b study of AADC gene therapy administered via a posterior approach
Objective: To present 3-year safety and clinical outcome data from a phase 1b, open-label trial of VY-AADC01 (NBIb-1817) for Parkinson’s disease (PD) patients with motor…
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