Eivind Nyland

@GreatStrides65 Also, the homozygous cohort result is additive on top of Symdeko (3-4% increase inFEV and a 10 decrease in mmol sweat chloride).

Here's the paper on other triple-combi: Headlines: 1. Absolute change in FEV1 of 13.8% for F508del/min heterozygous patients, 11.0% for F508del homozygous patients 2. Absolute reduction in sweat chloride of ~39 mmol/litre for both patient groups nejm.org/doi/full/10.10…

Here's the principal paper in the @NEJM on one of @VertexPharma's triple combis (Phase 2) nejm.org/doi/10.1056/NE…

@SymdekoT Tezacaftor (the other component in Symdeko) helps correct CFTR to enable it to reach the cell surface (which the DF508 mutation prevents). When at the cell surface, that protein acts like one with a gating defect (G551D) and Ivacaftor gets to do its thing.

Just wondering how Ivacaftor which targets mutation (G551D) is now magically working for DF508. Sounds like BS to me will have to research this more, this is a major component of Symdeko, but why use it if its not targeting my specific mutation???? #vertex #Symdeko

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