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Companies and Area of Research II-13
Gene Therapy- A Step Towards Cure II-14
PLASmin™ - The Only Investigational Drug Based on Gene Therapy II-14
Rising Concerns over Animal-Derived Drugs II-14
4. PIPELINE ANALYSIS II-15
Active Cystic Fibrosis Pipeline - A Ray of Hope II-15
Cystic Fibrosis Pipeline (As of January 2010) II-15
Gene Therapy II-15
Protein Assist/Repair Therapy: II-15
Restoration of Salt Transport Therapy: II-16
Anti-Inflammatory Agents: II-16
Anti-infective Agents: II-16
Other Developmental Drugs: II-17
5. DISEASE OVERVIEW II-18
Cystic Fibrosis - A Hereditary Disease II-18
Mutation in CFTR Gene - A Major Cause of CF II-18
DF508 - The Most Common Mutation that Affects CFTR Protein II-18
Table 2: Most Common Mutations and Worldwide Frequency
(includes corresponding Graph/Chart) II-18
Hereditary Risk of the Disease II-19
Pathophysiology of the Disease II-19
Symptoms of Cystic Fibrosis II-19
Common Symptoms in Early Childhood II-19
Common Symptoms in Grownups or Adults: II-20
Complications Associated with Cystic Fibrosis II-20
Sinus and Lung Illness II-20
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