J Lekstrom-Himes

TL;DR: These data show that lumacaftor in combination with ivacaftors provided a benefit for patients with cystic fibrosis homozygous for the Phe508del CFTR mutation.

TL;DR: CFTR modulator therapy with tezacaftor–ivacaftor or ivacaftors alone was efficacious in patients with cystic fibrosis who were heterozygous for the Phe508del deletion and a CFTR residual‐function mutation.

TL;DR: These results support continued clinical development of tezacaftor (100 mg every day) in combination with ivacaftor(150 mg every 12 h) in subjects with cystic fibrosis.

TL;DR: To induce potentially curative levels of HbF in erythrocytes, the ex vivo CRISPR-Cas9-based gene-editing platform was used to edit the erythroid enhancer region of BCL11A in hematopoietic stem and progenitor cells, producing CTX001.

TL;DR: Drug–drug interaction studies are described for tezacaftor/ivacaftor, a new cystic fibrosis transmembrane conductance regulator modulator therapy for the treatment of cystic Fibrosis, which is unlikely to impact exposure of drugs metabolized by CYP3A, including hormonal contraceptives.