CFTR corrector C17 is effective in muscular dystrophy, in vivo proof of concept in LGMDR3.
Scano M, Benetollo A, Nogara L, Bondì M, Dalla Barba F, Soardi M, Furlan S, Akyurek EE, Caccin P, Carotti M, Sacchetto R, Blaauw B, Sandonà D.
Scano M, et al. Among authors: blaauw b.
Hum Mol Genet. 2022 Feb 21;31(4):499-509. doi: 10.1093/hmg/ddab260.
Hum Mol Genet. 2022.
PMID: 34505136
Free PMC article.