© 2017

Treatment of Cystic Fibrosis and Other Rare Lung Diseases

  • Arata Azuma
  • Michael S. Schechter

Part of the Milestones in Drug Therapy book series (MDT)

Table of contents

  1. Front Matter
    Pages i-viii
  2. Etiopathology and Genetics of Rare Lung Diseases

    1. Front Matter
      Pages 1-1
    2. Nauman Chaudary, Michael S. Schechter
      Pages 3-20
    3. Naoto Keicho, Minako Hijikata
      Pages 21-38
    4. Chiko Shimbori, Pierre-Simon Bellaye, Philipp Kolb, Martin Kolb
      Pages 39-69
    5. Koh Nakata, Ryushi Tazawa
      Pages 71-86
    6. Kuniaki Seyama
      Pages 87-98
  3. Treatment of Cystic Fibrosis

    1. Front Matter
      Pages 99-99
    2. David R. Spielberg, John P. Clancy, Christopher Siracusa
      Pages 101-118
    3. Evangelia Daviskas, Sheila Sivam, Mark R. Elkins, Tiffany J. Dwyer, Ruth Dentice, Peter T. Bye
      Pages 119-138
    4. Elliott C. Dasenbrook, James F. Chmiel
      Pages 139-151
    5. Patrick A. Flume, Donald R. VanDevanter
      Pages 153-169
  4. Treatment of Other Rare Lung Diseases

    1. Front Matter
      Pages 171-171
    2. Mutsuo Yamaya, Arata Azuma, Shoji Kudoh
      Pages 173-188
    3. Paolo Spagnolo
      Pages 189-210
    4. Muhammad Muhye-ud-din Sheikh, Bruce C. Trapnell
      Pages 211-238
    5. Mariam Anis, Francis X. McCormack
      Pages 239-263

About this book


This volume describes the pathogenesis and pathophysiology of several pulmonary diseases as well as their treatment. It also discusses the underlying genetic and molecular biological basis, which opens the way for new treatments for these conditions.
It focuses on the treatment of cystic fibrosis including CFTR (cystic fibrosis transmembrane-conductance regulator) modulator therapies, drug therapies that augment airway surface liquid as well as anti-inflammatory and anti-infective therapies. Further topics include long-term, low-dose macrolide therapy for diffuse panbronchiolitis; novel agents for previously untreatable idiopathic pulmonary fibrosis; possible new treatments for pulmonary alveolar proteinosis (PAP); and multiple novel therapeutic targets for treating lymphangiomyomatosis. Research into these conditions has led to major advances in our understanding of the underlying genetic and molecular basis of this disease, and to dramatic improvements in survival and quality of life for affected individuals.


Pulmonary alveolar proteinosis Lymphangioleiomyomatosis Panbronchiolitis Idiopathic Pulmonary Fibrosis Interstitial pneumonia Mucociliary clearance Surfactant Orphan drugs Airway surface liquid

Editors and affiliations

  • Arata Azuma
    • 1
  • Michael S. Schechter
    • 2
  1. 1.Div of Pulmonary MedicineNippon Medical School Div of Pulmonary MedicineTokyoJapan
  2. 2.Children's Hospital of RichmondVirginia Commonwealth University Children's Hospital of RichmondRichmondUSA

Bibliographic information


“The purpose is to focus on recent advances in understanding the molecular basis of these diseases, and how this understanding has helped develop novel targeted therapies. … The audience is clinicians, students, and researchers who want a deeper understanding of the pathogenesis and recent advances in the therapy of these rare diseases. … It should serve as a useful addition to the personal library of any interested pulmonologist or lung disease researcher.” (Santosh Dhungana, Doody's Book Reviews, April, 2017)