Ontology highlight
ABSTRACT:
SUBMITTER: Thakkar N
PROVIDER: S-EPMC5613184 | biostudies-literature | 2017 Sep
REPOSITORIES: biostudies-literature
Thakkar Nilay N Guptill Jeffrey T JT Aleš Kathy K Jacobus David D Jacobus Laura L Peloquin Charles C Cohen-Wolkowiez Michael M Gonzalez Daniel D
CPT: pharmacometrics & systems pharmacology 20170724 9
Lambert-Eaton myasthenia (LEM) is a rare autoimmune disorder associated with debilitating muscle weakness. There are limited treatment options and 3,4-diaminopyridine (3,4-DAP) free base is an investigational orphan drug used to treat LEM-related weakness. We performed a population pharmacokinetic/pharmacodynamic (PK/PD) analysis using 3,4-DAP and metabolite concentrations collected from a phase II study in patients with LEM. The Triple Timed Up & Go (3TUG) assessment, which measures lower extre ...[more]