TY - JOUR
T1 - A Review of MD STARnet’s Research Contributions to Pediatric-Onset Dystrophinopathy in the United States; 2002-2017
AU - on behalf of the MD STARnet
AU - Sahay, Kashika M.
AU - Smith, Tiffany
AU - Conway, Kristin M.
AU - Romitti, Paul A.
AU - Lamb, Molly M.
AU - Andrews, Jennifer
AU - Pandya, Shree
AU - Oleszek, Joyce
AU - Cunniff, Christopher
AU - Valdez, Rodolfo
N1 - Funding Information:
The authors disclosed receipt of the following financial support for the research, authorship, and/or publication of this article: This publication was supported by the Cooperative Agreement numbers, DD000187, DD000189, DD000190, DD000191, DD000392, DD001126, DD001119, DD001123, funded by the Centers for Disease Control and Prevention. Its contents are solely the responsibility of the authors and do not necessarily represent the official views of the Centers for Disease Control and Prevention or the Department of Health and Human Services.
Publisher Copyright:
© The Author(s) 2018.
PY - 2019/1/1
Y1 - 2019/1/1
N2 - Population studies of rare disorders, such as Duchenne and Becker muscular dystrophies (dystrophinopathies), are challenging due to diagnostic delay and heterogeneity in disorder milestones. To address these challenges, the Centers for Disease Control and Prevention established the Muscular Dystrophy Surveillance, Tracking, and Research Network (MD STARnet) in 2002 in the United States. From 2002 to 2012, MD STARnet longitudinally tracked the prevalence, clinical, and health care outcomes of 1054 individuals born from 1982 to 2011 with pediatric-onset dystrophinopathy through medical record abstraction and survey data collection. This article summarizes 31 MD STARnet peer-reviewed publications. MD STARnet provided the first population-based prevalence estimates of childhood-onset dystrophinopathy in the United States. Additional publications provided insights into diagnostic delay, dystrophinopathy-specific growth charts, and health services use. Ongoing population-based surveillance continually improves our understanding of clinical and diagnostic outcomes of rare disorders.
AB - Population studies of rare disorders, such as Duchenne and Becker muscular dystrophies (dystrophinopathies), are challenging due to diagnostic delay and heterogeneity in disorder milestones. To address these challenges, the Centers for Disease Control and Prevention established the Muscular Dystrophy Surveillance, Tracking, and Research Network (MD STARnet) in 2002 in the United States. From 2002 to 2012, MD STARnet longitudinally tracked the prevalence, clinical, and health care outcomes of 1054 individuals born from 1982 to 2011 with pediatric-onset dystrophinopathy through medical record abstraction and survey data collection. This article summarizes 31 MD STARnet peer-reviewed publications. MD STARnet provided the first population-based prevalence estimates of childhood-onset dystrophinopathy in the United States. Additional publications provided insights into diagnostic delay, dystrophinopathy-specific growth charts, and health services use. Ongoing population-based surveillance continually improves our understanding of clinical and diagnostic outcomes of rare disorders.
KW - dystrophinopathy
KW - muscular dystrophy
KW - population-based
KW - public health surveillance
KW - rare disorders
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U2 - 10.1177/0883073818801704
DO - 10.1177/0883073818801704
M3 - Review article
C2 - 30345857
AN - SCOPUS:85058837279
VL - 34
SP - 44
EP - 53
JO - Journal of Child Neurology
JF - Journal of Child Neurology
SN - 0883-0738
IS - 1
ER -