A Review of MD STARnet’s Research Contributions to Pediatric-Onset Dystrophinopathy in the United States; 2002–2017

Details

• Alternative Title:
  J Child Neurol
• Personal Author:
  Sahay, Kashika M. ; Smith, Tiffany ; Conway, Kristin M. ; Romitti, Paul A. ; Lamb, Molly M. ; Andrews, Jennifer ; Pandya, Shree ; Oleszek, Joyce ; Cunniff, Christopher ; Valdez, Rodolfo
• Corporate Authors:
  on behalf of the MD STARnet
• Description:
  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.
• Subjects:
  Article Dystrophinopathy Muscular Dystrophy Population-based Public Health Surveillance Rare Disorders
• Source:
  J Child Neurol. 34(1):44-53
• Pubmed ID:
  30345857
• Pubmed Central ID:
  PMC6444919
• Document Type:
  Journal Article
• Funding:
  U01 DD000392/DD/NCBDD CDC HHS/United States ; U01 DD001126/DD/NCBDD CDC HHS/United States ; U01 DD000187/DD/NCBDD CDC HHS/United States ; U01 DD001123/DD/NCBDD CDC HHS/United States ; U01DD001123/ACL/ACL HHS/United States ; U01 DD000189/DD/NCBDD CDC HHS/United States ; U01DD001119/ACL/ACL HHS/United States ; CC999999/ImCDC/Intramural CDC HHS/United States ; U01DD001126/ACL/ACL HHS/United States ; U01 DD000191/DD/NCBDD CDC HHS/United States ; U01 DD001119/DD/NCBDD CDC HHS/United States ; U01 DD000190/DD/NCBDD CDC HHS/United States
• Volume:
  34
• Issue:
  1
• Collection(s):
  CDC Public Access
• Main Document Checksum:
  urn:sha-512:96dd16410490c1c3893a0799342ff70651f76f912e76c8f2f4368857aabe4a4e08e9d7e0d1308ad4753bf279184540cdd5ef73ecbf8b1a1371948d09c8fa3de0
• Download URL:
  https://stacks.cdc.gov/view/cdc/77176/cdc_77176_DS1.pdf
• File Type:
  [PDF - 619.35 KB ]