Simard, Christopher

Elexacaftor–Tezacaftor–Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele

Middleton, P. G., Mall, M. A., Dřevínek, P., Lands, L. C., McKone, E. F., Polineni, D., Ramsey, B. W., Taylor-Cousar, J. L., Tullis, E., Vermeulen, F., et al. (2019) · New England Journal of Medicine

Cystic Fibrosis Research AdvancesInhalation and Respiratory Drug Delivery

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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

Heijerman, H. G. M., McKone, E. F., Downey, D. G., Van Braeckel, E., Rowe, S. M., Tullis, E., Mall, M. A., Welter, J. J., Ramsey, B. W., McKee, C. M., et al. (2019) · The Lancet

Cystic Fibrosis Research AdvancesPharmacogenetics and Drug Metabolism

Cited by 1,414

Tezacaftor–Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del

Taylor-Cousar, J. L., Munck, A., McKone, E. F., van der Ent, C. K., Moeller, A., Simard, C., Wang, L. T., Ingenito, E. P., McKee, C., Lu, Y., et al. (2017) · New England Journal of Medicine

Cystic Fibrosis Research AdvancesInhalation and Respiratory Drug Delivery

Cited by 794

VX-445–Tezacaftor–Ivacaftor in Patients with Cystic Fibrosis and One or Two Phe508del Alleles

Keating, D., Marigowda, G., Burr, L., Daines, C., Mall, M. A., McKone, E. F., Ramsey, B. W., Rowe, S. M., Sass, L. A., Tullis, E., et al. (2018) · New England Journal of Medicine

Cystic Fibrosis Research AdvancesInhalation and Respiratory Drug Delivery

Cited by 726

Tezacaftor–Ivacaftor in Residual-Function Heterozygotes with Cystic Fibrosis

Rowe, S. M., Daines, C., Ringshausen, F. C., Kerem, E., Wilson, J., Tullis, E., Nair, N., Simard, C., Han, L., Ingenito, E. P., et al. (2017) · New England Journal of Medicine

Cystic Fibrosis Research AdvancesInhalation and Respiratory Drug Delivery

Cited by 522

Exagamglogene Autotemcel for Severe Sickle Cell Disease

Frangoul, H., Locatelli, F., Sharma, A., Bhatia, M., Mapara, M., Molinari, L., Wall, D., Liem, R. I., Telfer, P., Shah, A. J., et al. (2024) · New England Journal of Medicine

Hemoglobinopathies and Related DisordersVirus-based gene therapy research

Cited by 493

VX-659–Tezacaftor–Ivacaftor in Patients with Cystic Fibrosis and One or Two Phe508del Alleles

Davies, J. C., Moskowitz, S. M., Brown, C., Horsley, A., Mall, M. A., McKone, E. F., Plant, B. J., Prais, D., Ramsey, B. W., Taylor-Cousar, J. L., et al. (2018) · New England Journal of Medicine

Cystic Fibrosis Research AdvancesInhalation and Respiratory Drug Delivery

Cited by 331

Disease progression in patients with cystic fibrosis treated with ivacaftor: Data from national US and UK registries

Volkova, N., Moy, K., Evans, J., Campbell, D., Tian, S., Simard, C., Higgins, M., Konstan, M. W., Sawicki, G. S., Elbert, A., et al. (2020) · Journal of Cystic Fibrosis

Cystic Fibrosis Research AdvancesInhalation and Respiratory Drug Delivery

Cited by 258

Exagamglogene Autotemcel for Transfusion-Dependent β-Thalassemia

Locatelli, F., Lang, P., Wall, D., Meisel, R., Corbacioglu, S., Li, A. M., de la Fuente, J., Shah, A. J., Carpenter, B., Kwiatkowski, J. L., et al. (2024) · New England Journal of Medicine

Hemoglobinopathies and Related DisordersVirus-based gene therapy research

Cited by 253

Data from the US and UK cystic fibrosis registries support disease modification by CFTR modulation with ivacaftor

Bessonova, L., Volkova, N., Higgins, M., Bengtsson, L., Tian, S., Simard, C., Konstan, M. W., Sawicki, G. S., Sewall, A., Nyangoma, S., et al. (2018) · Thorax

Cystic Fibrosis Research AdvancesInhalation and Respiratory Drug Delivery

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