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Gene, RNA, and ASO-based therapeutic approaches in Cystic Fibrosis.
Allaire NE, Griesenbach U, Kerem B, Lueck JD, Stanleigh N, Oren YS. Allaire NE, et al. Among authors: kerem b. J Cyst Fibros. 2023 Mar;22 Suppl 1(Suppl 1):S39-S44. doi: 10.1016/j.jcf.2022.12.016. Epub 2023 Jan 17. J Cyst Fibros. 2023. PMID: 36658041 Free PMC article.
Most people with Cystic Fibrosis (PwCF) harbor Cystic Fibrosis Transmembrane Conductance (CFTR) mutations that respond to highly effective CFTR modulators (HEM); however, a small fraction of non-responsive variants will require alternative approaches for treatment. Furthermore, t …
Most people with Cystic Fibrosis (PwCF) harbor Cystic Fibrosis Transmembrane Conductance (CFTR) mutations that respond to highly effective C …
Effects of flecainide in patients with new SCN5A mutation: mutation-specific therapy for long-QT syndrome?
Benhorin J, Taub R, Goldmit M, Kerem B, Kass RS, Windman I, Medina A. Benhorin J, et al. Among authors: kerem b. Circulation. 2000 Apr 11;101(14):1698-706. doi: 10.1161/01.cir.101.14.1698. Circulation. 2000. PMID: 10758053
Flecainide also normalized the marked baseline repolarization dispersion in most mutation carriers. These effects among carriers were maintained during long-term (9 to 17 months) outpatient flecainide therapy with no adverse effects. ...
Flecainide also normalized the marked baseline repolarization dispersion in most mutation carriers. These effects among carriers were mainta …