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Clinical evaluation of VX-809, a novel investigational oral F508del-CFTR corrector, in subjects with cystic fibrosis homozygous for the F508del–CFTR mutation

✍ Scribed by J.P. Clancy; G. Spencer-Green


Book ID
118642513
Publisher
Elsevier Science
Year
2010
Tongue
English
Weight
52 KB
Volume
9
Category
Article
ISSN
1569-1993

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Cystic fibrosis patients with mutation 1
✍ V. Nunes; T. Casals; A. Gaona; G. Antiñolo; J. Ferrer-Calvete; J. Pérez-Frias; E 📂 Article 📅 1992 🏛 John Wiley and Sons 🌐 English ⚖ 437 KB

The majority of the identified cystic fibrosis (CF) mutations are very uncommon in the total patient population, making the correlation between the clinical presentation and the molecular alterations difficult. The largest deletion that has been described so far in C F is of 84 bp in exon 13, which