LRRK2 PROTAC® degrader molecules induce robust biomarker responses in preclinical in vivo pharmacology studies following acute and chronic oral dosing
Objective: Report on the non-clinical in vivo pharmacokinetic and pharmacodynamic biomarker response to PROteolysis TArgeting Chimera (PROTAC®) molecules designed to induce degradation of leucine rich…Development of potent, orally bioavailable, and highly selective LRRK2 PROTAC® degrader molecules as potential disease modifying therapeutics for Parkinson’s disease
Objective: Report on the pharmacological characteristics of small molecule PROteolysis TArgeting Chimera (PROTAC) molecules designed to induce degradation of leucine rich repeat kinase 2 (LRRK2)…Consanguinity is the key player in complexity of Mendelian form of neurodegeneration with brain iron accumulation in Pakistan
Objective: In the current work, we recognized big inbreed Pakistani family with autosomal recessive NBIA containing several affected individuals. Aim of the research work was…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…
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