Cystic fibrosis remains one of the most common fatal genetic diseases, as well as the most common fatal hereditary lung disease worldwide. In recent years, medical scientists have made intensified attempts at understanding the disease mechanism at the core of cystic fibrosis, namely the defect in the CFTR protein. In this regard, Marcus Mall and his team have made groundbreaking contributions. A specialist in paediatric pulmonology and physician-scientist, Mall is the Director at the Department of Pediatric Respiratory Medicine, Immunology and Critical Care Medicine at Charité – Universitätsmedizin Berlin. He led the first clinical trial that demonstrated that a combination therapy of three small molecules leads to unprecedented improvement in lung function in patients affected by cystic fibrosis. At Falling Walls, Mall discusses how he and his team contributed to unraveling of disease mechanisms underlying cystic fibrosis and how the development of a highly effective therapy that restores function of the CFTR protein can improve the quality of life in the majority of patients with this devastating disease.

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