Rare Diseases



Cystic Fibrosis

By acquiring Profile Pharma in the UK in 2013, Zambon starts building a product portfolio for rare diseases, especially for cystic fibrosis.

Cystic fibrosis is the most common of all severe genetic diseases. There are about 100,000 people affected by the disease in the world. Progress made by research and treatment has ensured children who are born with the disease today a mean life expectancy of 40 years and more, while they did not go beyond infancy fifty years ago, when the disease was first discovered and early treatment was attempted.

Cystic fibrosis produces thick secretions that especially damage the respiratory and digestive system. Bronchi and lungs present recurrent bronchitis and bronchial pneumonia caused by particular bacteria, especially Pseudomonas aeruginosa and Staphylococcus aureus. The persistence of these bacteria causes chronic infection and inflammation in the lungs with their subsequent deterioration and gradual decline of respiratory function leading to respiratory failure.

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