• Dtsch. Med. Wochenschr. · Oct 2020

    [Therapy of cystic fibrosis - new drugs give hope].

    • Patience Eschenhagen and Carsten Schwarz.
    • Sektion Cystische Fibrose, Christiane Herzog-Zentrum Berlin, Charité - Universitätsmedizin Berlin, Klinik für Pädiatrie m. S. Pneumologie, Immunologie und Intensivmedizin, Campus Rudolf Virchow Klinikum, Berlin.
    • Dtsch. Med. Wochenschr. 2020 Oct 1; 145 (20): 1486-1489.

    AbstractFor decades there were only symptom-oriented therapies for the monogenetic disease cystic fibrosis. With the new modulator therapies there are new hopes to better influence the course of the disease, which has its manifestation in several organs such as the lungs, the liver or pancreas.In addition to ivacaftor, lumacaftor/ivacaftor and tezacaftor/ivacaftor, a triple combination with elexacaftor/tezacaftor/ivacaftor was developed. Studies in F508del homozygous and heterozygous patients with cystic fibrosis have resulted in significant clinical improvement and have therefore been approved by the FDA for the first time in the USA.The results are very promising as already after 4 weeks a significant improvement of FEV1 and a significant decrease of the sweat chloride content could be detected. In the USA all patients with at least one F508del mutation can be prescribed the drug. This corresponds to approximately 90 % of all patients with cystic fibrosis and has the potential to change the landscape of cystic fibrosis.Thieme. All rights reserved.

      Pubmed     Full text   Copy Citation     Plaintext  

      Add institutional full text...

    Notes

     
    Knowledge, pearl, summary or comment to share?
    300 characters remaining
    help        
    You can also include formatting, links, images and footnotes in your notes
    • Simple formatting can be added to notes, such as *italics*, _underline_ or **bold**.
    • Superscript can be denoted by <sup>text</sup> and subscript <sub>text</sub>.
    • Numbered or bulleted lists can be created using either numbered lines 1. 2. 3., hyphens - or asterisks *.
    • Links can be included with: [my link to pubmed](http://pubmed.com)
    • Images can be included with: ![alt text](https://bestmedicaljournal.com/study_graph.jpg "Image Title Text")
    • For footnotes use [^1](This is a footnote.) inline.
    • Or use an inline reference [^1] to refer to a longer footnote elseweher in the document [^1]: This is a long footnote..

    hide…