Newborn screening for cystic fibrosis.
CF newborn screening benefits growth and prevents malnutrition in children with CF. Increasing evidence suggests long-term pulmonary benefits, but these data are still accumulating. With a standardized approach to clinical care for these pre-symptomatic patients, we should be able to ask questions about the optimal treatment strategy for therapies that are currently available, perhaps through randomized clinical trials in this group. For developing therapies, Massachusetts will have identified its CF patients before they have sustained significant malnutrition and lung injury, and it may be these patients who will benefit most from therapies still on the horizon.
Parad
Richard B
RB
Massachusetts Cystic Fibrosis Newborn Screening Program, University of Massachusetts Medical School, Department of Pediatrics, Harvard Medical School, Brigham and Women's Hospital, Children's Hospital, Boston, USA.
Comeau
Anne Marie
AM
D003365:Costs and Cost Analysis; D003550:Cystic Fibrosis / Q000175:diagnosis* / Q000453:epidemiology / Q000235:genetics; D005820:Genetic Testing; D006801:Humans; D007231:Infant, Newborn; D015997:Neonatal Screening / Q000191:economics; D014481:United States