Journal article
A Review of MD STARnet’s Research Contributions to Pediatric-Onset Dystrophinopathy in the United States; 2002–2017
Journal of child neurology, Vol.34(1), pp.44-53
01/2019
DOI: 10.1177/0883073818801704
PMCID: PMC6444919
PMID: 30345857
Abstract
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 STAR
net
) in 2002 in the United States. From 2002 to 2012, MD STAR
net
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 STAR
net
peer-reviewed publications. MD STAR
net
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.
Details
- Title: Subtitle
- A Review of MD STARnet’s Research Contributions to Pediatric-Onset Dystrophinopathy in the United States; 2002–2017
- Creators
- Kashika M Sahay - Centers for Disease Control and Prevention, National Center for Birth Defects and Developmental Disabilities, DHDD, Rare Disorders and Health Outcomes team, Atlanta, GA, USATiffany Smith - Centers for Disease Control and Prevention, National Center for Birth Defects and Developmental Disabilities, DHDD, Rare Disorders and Health Outcomes team, Atlanta, GA, USAKristin M Conway - Centers for Disease Control and Prevention, National Center for Birth Defects and Developmental Disabilities, DHDD, Rare Disorders and Health Outcomes team, Atlanta, GA, USAPaul A Romitti - Centers for Disease Control and Prevention, National Center for Birth Defects and Developmental Disabilities, DHDD, Rare Disorders and Health Outcomes team, Atlanta, GA, USAMolly M Lamb - Centers for Disease Control and Prevention, National Center for Birth Defects and Developmental Disabilities, DHDD, Rare Disorders and Health Outcomes team, Atlanta, GA, USAJennifer Andrews - Centers for Disease Control and Prevention, National Center for Birth Defects and Developmental Disabilities, DHDD, Rare Disorders and Health Outcomes team, Atlanta, GA, USAShree Pandya - Centers for Disease Control and Prevention, National Center for Birth Defects and Developmental Disabilities, DHDD, Rare Disorders and Health Outcomes team, Atlanta, GA, USAJoyce Oleszek - Centers for Disease Control and Prevention, National Center for Birth Defects and Developmental Disabilities, DHDD, Rare Disorders and Health Outcomes team, Atlanta, GA, USAChristopher Cunniff - Centers for Disease Control and Prevention, National Center for Birth Defects and Developmental Disabilities, DHDD, Rare Disorders and Health Outcomes team, Atlanta, GA, USARodolfo Valdez - Centers for Disease Control and Prevention, National Center for Birth Defects and Developmental Disabilities, DHDD, Rare Disorders and Health Outcomes team, Atlanta, GA, USAMD STARnet
- Resource Type
- Journal article
- Publication Details
- Journal of child neurology, Vol.34(1), pp.44-53
- DOI
- 10.1177/0883073818801704
- PMID
- 30345857
- PMCID
- PMC6444919
- ISSN
- 0883-0738
- eISSN
- 1708-8283
- Grant note
- DOI: 10.13039/100000030, name: Centers for Disease Control and Prevention, award: DD001119
- Language
- English
- Date published
- 01/2019
- Academic Unit
- Epidemiology; Biostatistics
- Record Identifier
- 9983995123502771
Metrics
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