학술논문

A novel, hybrid, single- and multi-site clinical trial design for CLN3 disease, an ultra-rare lysosomal storage disorder.
Document Type
Article
Source
Clinical Trials; Oct2019, Vol. 16 Issue 5, p555-560, 6p, 1 Chart, 2 Graphs
Subject
MYCOPHENOLIC acid
CLINICAL trials
COMPARATIVE studies
CROSSOVER trials
DRUG delivery systems
EXPERIMENTAL design
GENES
LYSOSOMAL storage diseases
MATHEMATICAL models
PATIENT-professional relations
GENETIC mutation
NERVOUS system
NEURODEGENERATION
NEURONAL ceroid-lipofuscinosis
PATIENT monitoring
PATIENT safety
PEDIATRICS
PLACEBOS
TIME
TRAVEL
THEORY
TREATMENT effectiveness
HUMAN services programs
HUMAN research subjects
DESCRIPTIVE statistics
EVALUATION
THERAPEUTICS
Language
ISSN
17407745
Abstract
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