Treatment of Cystic Fibrosis and Other Rare Lung Diseases (Milestones in Drug Therapy)

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. Front Matter....Pages i-viii Front Matter....Pages 1-1 An Introduction to Clinical Aspects of Cystic Fibrosis....Pages 3-20 Diffuse Panbronchiolitis....Pages 21-38 Idiopathic Pulmonary Fibrosis....Pages 39-69 Pulmonary Alveolar Proteinosis: A Historic Perspective....Pages 71-86 Lymphangioleiomyomatosis....Pages 87-98 Front Matter....Pages 99-99 CFTR Modulator Therapies in Cystic Fibrosis....Pages 101-118 Drug Therapies that Augment Airway Surface Liquid....Pages 119-138 Anti-Inflammatory Therapies for Cystic Fibrosis....Pages 139-151 Anti-Infective Therapies in Cystic Fibrosis....Pages 153-169 Front Matter....Pages 171-171 Diffuse Panbronchiolitis: Long-Term Low-Dose Macrolide Therapy....Pages 173-188 Idiopathic Pulmonary Fibrosis....Pages 189-210 Treatment of Pulmonary Alveolar Proteinosis....Pages 211-238 Treatment of Lymphangioleiomyomatosis (LAM)....Pages 239-263 Arata Azuma, Michael S. Schechter, Editors. Includes Bibliographical References.