Last Posted: Nov 03, 2019
- Efficacy and safety of the elexacaftor plus tezacaftor plus ivacaftor combination regimen in people with cystic fibrosis homozygous for the F508del mutation: a double-blind, randomised, phase 3 trial
HGM Heijerman et al, Lancet, October 31, 2019 - Elexacaftor–Tezacaftor–Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele
PJ Middleton et al, NEJM, October 31, 2019 - FDA approves new breakthrough therapy for cystic fibrosis- Treatment approved for approximately 90% of patients with cystic fibrosis, many of whom had no approved therapeutic options
FDA News Release, October 2019 - Studies Yield ‘Impressive’ Results in Fight Against Cystic Fibrosis
N Chokshi, NY Times, October 31, 2019 - New Hope For Patients Living With Cystic Fibrosis After Scientists Unveil Therapy
NPR, November 1, 2019 - Dare to Dream: The Long Road to Targeted Therapies for Cystic Fibrosis
NIH Director's Blog, October 31, 2019 - 22q and two: 22q11.2 deletion syndrome and coexisting conditions.
Cohen Jennifer L et al. American journal of medical genetics. Part A 2018 176(10) 2203-2214 - Whole Blood Gene Expression Profiling Predicts Severe Morbidity and Mortality in Cystic Fibrosis: A 5-Year Follow-Up Study.
Saavedra Milene T et al. Annals of the American Thoracic Society 2018 15(5) 589-598 - Immunoreactive trypsinogen levels in newborn screened infants with an inconclusive diagnosis of cystic fibrosis.
Ooi Chee Y et al. BMC pediatrics 2019 Oct 19(1) 369 - Factors affecting the growth of infants diagnosed with cystic fibrosis by newborn screening.
Patterson K D et al. BMC pediatrics 2019 Oct 19(1) 356
.png)
No hay comentarios:
Publicar un comentario