Explores preclinical and clinical trials in SMA gene therapy, including efficiency in patients, trial results, adverse effects, cellular targeting, and future perspectives.
Explores the selection and rationale of preclinical animal models in drug development, emphasizing the 10 to 15-year path to a marketed small-molecule drug.
Explores kinase inhibitors, enzyme inhibition methods, transition state analog drugs, and the significance of covalent drugs in pharmaceutical research.
Explores kinase inhibitors, their types, mechanisms, and experimental evaluation, emphasizing the significance of covalent drugs in modern pharmaceutical research.
Explores the network effects of deep brain stimulation beyond the basal ganglia, focusing on optimal connectivity and the role of brain oscillations in PD motor symptoms.
Explores the selection and rationale behind preclinical animal models in drug discovery, emphasizing understanding mechanisms of action and domain specificity.
