Article
Advances in the diagnosis and treatment of cystic fibrosis.
Advances in pediatrics - 1 Aug 2014
Martiniano Stacey L, Hoppe Jordana E, Sagel Scott D, Zemanick Edith T
Abstract excerpt
CF is a genetic, life-shortening, multisystem disease that is most commonly diagnosed through newborn screen performed in all 50 states in the United States. In the past, therapies for CF lung disease have primarily targeted the downstream effects of a dysfunctional CFTR protein. Newer CFTR modulator therapies, targeting the basic defect in CF, are available for a limited group of people with CF, and offer the...
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