Table 1.
Therapies for cystic fibrosis
| Treatment of Clinical Consequences | |
| 1. Pancreatic Disease | • Pancreatic enzyme replacement (PERT) |
| 2. Lung Disease | |
| a) Mucus clearance | • DNAse - Pulmozyme (Genentech) |
| • UTP analog - Donufosol (Inspire Pharmaceuticals) | |
| • Lantibiotic - Duramycin (Moli 1901 - Lantibio) | |
| • Hypertonic saline | |
| b) Anti-inflammation | • Nonsteroidal anti-inflammatory drugs - Ibuprofen |
| • Corticosteroids - Prednisone | |
| • Macrolide antibiotics - Zithromax | |
| c) Anti-microbial | • TOBI (Novartis) - for P.aeruginosa |
| Gene Variant Targeted Pharmaceutical Compounds (Personalized Treatment) | |
| Protein truncation | • Aminoglycoside antibiotics - Gentamicin |
| • Non-aminoglycoside compounds - PTC124 (Ataluren) (PTC Therapeutics) | |
| Channel regulation (Potentiators) | • Vx-770 (Vertex pharmaceuticals) - G551D Mutation |
| Protein folding (Correctors) | • Vx-809 (Vertex pharmaceuticals) - ΔF508 Mutation |