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Ataluren (PTC124) Induces Cystic Fibrosis Transmembrane Conductance Regulator Protein Expression and Activity in Children with Nonsense Mutation Cystic Fibrosis

DOI

240

Citations

38

References

2010

Year

Isabelle Sermet‐Gaudelus, K. De Boeck, Georges Casimir, F. Vermeulen, Teresinha Leal, Agnès Mogenet, Delphine Roussel, Janine Fritsch, L. Hanssens, Samit Hirawat,
American Journal of Respiratory and Critical Care Medicine

Abstract

In children with nonsense mutation CF, ataluren can induce functional CFTR production and is well tolerated.

References

YearCitations

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