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cystic fibrosis

This page shows the latest cystic fibrosis news and features for those working in and with pharma, biotech and healthcare.

Vertex continues upward trajectory with French Orkambi deal

Vertex continues upward trajectory with French Orkambi deal

Orkambi (lumacaftor/ivacaftor) has been approved for national reimbursement in France for people aged two or over with cystic fibrosis, who have two copies of the F508del mutation in the CTFR ... Importantly, children with cystic fibrosis as young as two

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  • Growing up as a patient is hard to manage Growing up as a patient is hard to manage

    Given that many treatment regimens, such as those for diabetes or cystic fibrosis, require meal-time medication to be taken at school, it is easy to understand how the perceived pressure

  • Evolution or revolution: the latest review of NICE methods Evolution or revolution: the latest review of NICE methods

    Those who gave evidence to the APPG included staff from the Cystic Fibrosis Trust, Takeda, AstraZeneca, EMIG and FIECON, as well as from NICE.

  • The fight for affordable insulin The fight for affordable insulin

    Dialogue with pharma? No thanks. As with the UK’s Cystic Fibrosis Buyers’ Club advocates, T1International doesn’t see dialogue with pharma as being worthwhile – after years of frustration and debates

  • The Cystic Fibrosis Buyers’ Club The Cystic Fibrosis Buyers’ Club

    to source what could also be life- saving drugs: the Cystic Fibrosis Buyers’ Club. ... However, while it’s an alternative for those affected by cystic fibrosis, it’s by no means the most ideal one.

  • Vertex: a vision of treating all CF patients worldwide draws closer Vertex: a vision of treating all CF patients worldwide draws closer

    Luckily, that plan B was waiting in the wings: Kalydeco (ivacaftor), the first ever disease-modifying treatment for cystic fibrosis gained approval in 2012. ... As many commentators have pointed out, early research into the disease was only sustained

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