New and evolving therapies for cystic fibrosis patients

Alisha Jabar; Hengameh H. Raissy; Kathryn Blake

doi:10.1089/ped.2014.0364

New and evolving therapies for cystic fibrosis patients

Alisha Jabar
, Hengameh H. Raissy
, Kathryn Blake

University of New Mexico
Alfred I. duPont Hospital for Children

Research output: Contribution to journal › Article › peer-review

Abstract

Cystic fibrosis (CF) is a common autosomal recessive inherited disorder affecting 1 in 2,500 births and ∼75,000 people in North America, Europe, and Australia. The purpose of this review is to discuss the most recent advances in therapies for CF patients. The CF drug development pipeline provides information regarding the phase of each new therapy and those that are already in use by patients. The ultimate goal of therapy will be to deliver tailored individualized therapies to those who need it the most. This review will focus on new research on CF transmembrane conductance regulator protein modulator agents and the effort of gene therapy.

Original language	English
Pages (from-to)	92-94
Number of pages	3
Journal	Pediatric, Allergy, Immunology, and Pulmonology
Volume	27
Issue number	2
DOIs	https://doi.org/10.1089/ped.2014.0364
State	Published - 1 Jun 2014
Externally published	Yes

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10.1089/ped.2014.0364

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abstract = "Cystic fibrosis (CF) is a common autosomal recessive inherited disorder affecting 1 in 2,500 births and ∼75,000 people in North America, Europe, and Australia. The purpose of this review is to discuss the most recent advances in therapies for CF patients. The CF drug development pipeline provides information regarding the phase of each new therapy and those that are already in use by patients. The ultimate goal of therapy will be to deliver tailored individualized therapies to those who need it the most. This review will focus on new research on CF transmembrane conductance regulator protein modulator agents and the effort of gene therapy.",

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AB - Cystic fibrosis (CF) is a common autosomal recessive inherited disorder affecting 1 in 2,500 births and ∼75,000 people in North America, Europe, and Australia. The purpose of this review is to discuss the most recent advances in therapies for CF patients. The CF drug development pipeline provides information regarding the phase of each new therapy and those that are already in use by patients. The ultimate goal of therapy will be to deliver tailored individualized therapies to those who need it the most. This review will focus on new research on CF transmembrane conductance regulator protein modulator agents and the effort of gene therapy.

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New and evolving therapies for cystic fibrosis patients

Abstract

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