Measuring Health-Related Quality of Life in Tuberous Sclerosis Complex – Psychometric Evaluation of Three Instruments in Individuals With Refractory Epilepsy

Patient-Reported Outcome and Observer-Reported Outcome Assessment in Rare Disease Clinical Trials: An ISPOR COA Emerging Good Practices Task Force Report

(2017) Katy Benjamin et al.   VALUE IN HEALTH

Adjunctive everolimus therapy for treatment-resistant focal-onset seizures associated with tuberous sclerosis (EXIST-3): a phase 3, randomised, double-blind, placebo-controlled study

(2016) Jacqueline A French et al.   LANCET

Tuberous sclerosis complex

(2016) Elizabeth P. Henske et al.   Nature Reviews Disease Primers

Neurological and neuropsychiatric aspects of tuberous sclerosis complex

(2015) Paolo Curatolo et al.   LANCET NEUROLOGY

Tuberous Sclerosis Associated Neuropsychiatric Disorders (TAND) and the TAND Checklist

(2015) Petrus J. de Vries et al.   PEDIATRIC NEUROLOGY

Pediatric Patient-Reported Outcome Instruments for Research to Support Medical Product Labeling: Report of the ISPOR PRO Good Research Practices for the Assessment of Children and Adolescents Task Force

(2013) Louis S. Matza et al.   VALUE IN HEALTH

Defining minimally important change in QOLIE-31 scores: Estimates from three placebo-controlled lacosamide trials in patients with partial-onset seizures

(2012) Simon Borghs et al.   EPILEPSY & BEHAVIOR

Recommended methods for determining responsiveness and minimally important differences for patient-reported outcomes

(2007) Dennis Revicki et al.   JOURNAL OF CLINICAL EPIDEMIOLOGY