科研之友
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摘要
Cystic fibrosis (CF) is a common autosomal recessive inherited disorder affecting 1 in 2,500 births and similar to 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.
- 出版日期2014-6
