TY - JOUR
T1 - Current methodological issues in the study of children with inherited neuromuscular disorders
AU - Mercuri, Eugenio Maria
AU - Messina, Sonia
AU - Pane, Marika
AU - Bertini, Enrico
AU - Bertini, Enrico Silvio
PY - 2008
Y1 - 2008
N2 - Several clinical trials assessing children with hereditary neuromuscular disorders have been performed over the last decade. These studies highlighted issues related to design and performance of clinical studies assessing children with this group of disorders. This article reviews recent literature and clinical experience in this area, highlighting methodological shortcomings and disease-specific clinical confounding factors that should be considered for future study design. The state of the art of outcome measures in neuromuscular disorders will be discussed, and suggestions for future clinical trials are presented. © 2008 Blackwell Publishing Ltd.
AB - Several clinical trials assessing children with hereditary neuromuscular disorders have been performed over the last decade. These studies highlighted issues related to design and performance of clinical studies assessing children with this group of disorders. This article reviews recent literature and clinical experience in this area, highlighting methodological shortcomings and disease-specific clinical confounding factors that should be considered for future study design. The state of the art of outcome measures in neuromuscular disorders will be discussed, and suggestions for future clinical trials are presented. © 2008 Blackwell Publishing Ltd.
KW - neuromuscolar disorders
KW - neuromuscolar disorders
UR - http://hdl.handle.net/10807/260310
U2 - 10.1111/j.1469-8749.2008.02066.x
DO - 10.1111/j.1469-8749.2008.02066.x
M3 - Article
SN - 0012-1622
VL - 50
SP - 417
EP - 421
JO - Developmental Medicine and Child Neurology
JF - Developmental Medicine and Child Neurology
ER -