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…Impact of rs6265 in the Host and Donor on Dopamine Neuron Graft Function.
Objective: Extend investigations into the effect of the rs6265 single nucleotide polymorphism (SNP) on dopamine (DA) neuron graft efficacy using a parkinsonian rat model. Background:…Is there a role for safinamide in atypical parkinsonian syndromes?
Objective: To search for possible benefits of safinamide in atypical parkinsonism. Background: Safinamide is a selective reversible monoamine oxidase-B inhibitor with both dopaminergic and glutamatergic…Neurophysiological and Clinical Effects of Low-intensity Transcranial Ultrasound of the Motor Cortex in Parkinson’s Disease
Objective: Evaluate the neurophysiological and clinical effects of transcranial ultrasound stimulation (TUS) of motor cortex (M1) in PD patients. Background: Low-intensity TUS is a novel,…Longitudinal Program to Prevent PD (LoPP-PD): Multimodal Risk Stratification Informs Personalized Intervention
Objective: Longitudinal evaluation of personalized intervention strategies informed by detailed clinical and laboratory biomarker analysis to reduce relative risk of Parkinson’s disease in first degree…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.…
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