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- [1] Results of a phase IIa study of VX-809, an investigational CFTR corrector compound, in subjects with cystic fibrosis homozygous for the F508del-CFTR mutation[J]. THORAX, 2012, 67 (01) : 12 - 18Clancy, J. P.论文数: 0 引用数: 0 h-index: 0机构: Cincinnati Childrens Hosp, Med Ctr, Dept Pediat, Cincinnati, OH 45229 USA Cincinnati Childrens Hosp, Med Ctr, Dept Pediat, Cincinnati, OH 45229 USARowe, Steven M.论文数: 0 引用数: 0 h-index: 0机构: Cincinnati Childrens Hosp, Med Ctr, Dept Pediat, Cincinnati, OH 45229 USAAccurso, Frank J.论文数: 0 引用数: 0 h-index: 0机构: Univ Alabama, Dept Med, Birmingham, AL 35294 USA Cincinnati Childrens Hosp, Med Ctr, Dept Pediat, Cincinnati, OH 45229 USAAitken, Moira L.论文数: 0 引用数: 0 h-index: 0机构: Univ Colorado, Hlth Sci Ctr, Dept Pediat, Denver, CO 80262 USA Cincinnati Childrens Hosp, Med Ctr, Dept Pediat, Cincinnati, OH 45229 USAAmin, Raouf S.论文数: 0 引用数: 0 h-index: 0机构: Cincinnati Childrens Hosp, Med Ctr, Dept Pediat, Cincinnati, OH 45229 USA Cincinnati Childrens Hosp, Med Ctr, Dept Pediat, Cincinnati, OH 45229 USAAshlock, Melissa A.论文数: 0 引用数: 0 h-index: 0机构: Univ Washington, Dept Med, Div Pulm & Crit Care, Seattle, WA USA Cincinnati Childrens Hosp, Med Ctr, Dept Pediat, Cincinnati, OH 45229 USABallmann, Manfred论文数: 0 引用数: 0 h-index: 0机构: Cyst Fibrosis Fdn, Bethesda, MD USA Cincinnati Childrens Hosp, Med Ctr, Dept Pediat, Cincinnati, OH 45229 USABoyle, Michael P.论文数: 0 引用数: 0 h-index: 0机构: Hannover Med Sch, Dept Pediat, D-3000 Hannover, Germany Johns Hopkins Univ, Sch Med, Johns Hopkins Adult Cyst Fibrosis Program, Baltimore, MD USA Cincinnati Childrens Hosp, Med Ctr, Dept Pediat, Cincinnati, OH 45229 USABronsveld, Inez论文数: 0 引用数: 0 h-index: 0机构: Univ Med Ctr Utrecht, Dept Pulmonol & TB, Utrecht, Netherlands Cincinnati Childrens Hosp, Med Ctr, Dept Pediat, Cincinnati, OH 45229 USACampbell, Preston W.论文数: 0 引用数: 0 h-index: 0机构: Univ Washington, Dept Med, Div Pulm & Crit Care, Seattle, WA USA Cincinnati Childrens Hosp, Med Ctr, Dept Pediat, Cincinnati, OH 45229 USADe Boeck, Kris论文数: 0 引用数: 0 h-index: 0机构: Katholieke Univ Leuven, Louvain, Belgium Cincinnati Childrens Hosp, Med Ctr, Dept Pediat, Cincinnati, OH 45229 USADonaldson, Scott H.论文数: 0 引用数: 0 h-index: 0机构: Univ N Carolina, Cyst Fibrosis Pulm Res & Treatment Ctr, Chapel Hill, NC USA Cincinnati Childrens Hosp, Med Ctr, Dept Pediat, Cincinnati, OH 45229 USADorkin, Henry L.论文数: 0 引用数: 0 h-index: 0机构: Childrens Hosp, Div Pulm Med, Boston, MA 02115 USA Cincinnati Childrens Hosp, Med Ctr, Dept Pediat, Cincinnati, OH 45229 USADunitz, Jordan M.论文数: 0 引用数: 0 h-index: 0机构: Univ Minnesota, Sch Med, Dept Med, Minnesota Cyst Fibrosis Ctr, Minneapolis, MN 55455 USA Cincinnati Childrens Hosp, Med Ctr, Dept Pediat, Cincinnati, OH 45229 USADurie, Peter R.论文数: 0 引用数: 0 h-index: 0机构: Univ Toronto, Hosp Sick Children, Res Inst, Toronto, ON M5G 1X8, Canada Univ Toronto, Dept Pediat, Toronto, ON M5G 1X8, Canada Cincinnati Childrens Hosp, Med Ctr, Dept Pediat, Cincinnati, OH 45229 USAJain, Manu论文数: 0 引用数: 0 h-index: 0机构: Northwestern Univ, Feinberg Sch Med, Div Pulm & Crit Care Med, Chicago, IL 60611 USA Cincinnati Childrens Hosp, Med Ctr, Dept Pediat, Cincinnati, OH 45229 USALeonard, Anissa论文数: 0 引用数: 0 h-index: 0机构: Catholic Univ Louvain, Div Pediat Pulmonol, B-1200 Brussels, Belgium Cincinnati Childrens Hosp, Med Ctr, Dept Pediat, Cincinnati, OH 45229 USAMccoy, Karen S.论文数: 0 引用数: 0 h-index: 0机构: Nationwide Childrens Hosp, Columbus, OH USA Cincinnati Childrens Hosp, Med Ctr, Dept Pediat, Cincinnati, OH 45229 USAMoss, Richard B.论文数: 0 引用数: 0 h-index: 0机构: Stanford Univ, Sch Med, Dept Pediat, Stanford, CA 94305 USA Cincinnati Childrens Hosp, Med Ctr, Dept Pediat, Cincinnati, OH 45229 USAPilewski, Joseph M.论文数: 0 引用数: 0 h-index: 0机构: Univ Pittsburgh, Div Pulm Allergy & Crit Care Med, Pittsburgh, PA USA Cincinnati Childrens Hosp, Med Ctr, Dept Pediat, Cincinnati, OH 45229 USARosenbluth, Daniel B.论文数: 0 引用数: 0 h-index: 0机构: Washington Univ, Sch Med, Div Pulm & Crit Care Med, St Louis, MO USA Cincinnati Childrens Hosp, Med Ctr, Dept Pediat, Cincinnati, OH 45229 USARubenstein, Ronald C.论文数: 0 引用数: 0 h-index: 0机构: Univ Penn, Sch Med, Childrens Hosp Philadelphia, Div Pulm Med, Philadelphia, PA 19104 USA Univ Penn, Sch Med, Childrens Hosp Philadelphia, Cyst Fibrosis Ctr, Philadelphia, PA 19104 USA Univ Penn, Sch Med, Dept Pediat, Philadelphia, PA 19104 USA Emory Univ, Dept Pediat, Atlanta, GA 30322 USA Childrens Healthcare Atlanta, Atlanta, GA 30322 USA Cincinnati Childrens Hosp, Med Ctr, Dept Pediat, Cincinnati, OH 45229 USASchechter, Michael S.论文数: 0 引用数: 0 h-index: 0机构: Emory Univ, Dept Pediat, Atlanta, GA 30322 USA Childrens Healthcare Atlanta, Atlanta, GA 30322 USA Cincinnati Childrens Hosp, Med Ctr, Dept Pediat, Cincinnati, OH 45229 USABotfield, Martyn论文数: 0 引用数: 0 h-index: 0机构: Vertex Pharmaceut Inc, Cambridge, MA USA Cincinnati Childrens Hosp, Med Ctr, Dept Pediat, Cincinnati, OH 45229 USAOrdonez, Claudia L.论文数: 0 引用数: 0 h-index: 0机构: Vertex Pharmaceut Inc, Cambridge, MA USA Cincinnati Childrens Hosp, Med Ctr, Dept Pediat, Cincinnati, OH 45229 USASpencer-Green, George T.论文数: 0 引用数: 0 h-index: 0机构: Vertex Pharmaceut Inc, Cambridge, MA USA Cincinnati Childrens Hosp, Med Ctr, Dept Pediat, Cincinnati, OH 45229 USAVernillet, Laurent论文数: 0 引用数: 0 h-index: 0机构: Vertex Pharmaceut Inc, Cambridge, MA USA Cincinnati Childrens Hosp, Med Ctr, Dept Pediat, Cincinnati, OH 45229 USAWisseh, Steve论文数: 0 引用数: 0 h-index: 0机构: Vertex Pharmaceut Inc, Cambridge, MA USA Cincinnati Childrens Hosp, Med Ctr, Dept Pediat, Cincinnati, OH 45229 USAYen, Karl论文数: 0 引用数: 0 h-index: 0机构: Vertex Pharmaceut Inc, Cambridge, MA USA Cincinnati Childrens Hosp, Med Ctr, Dept Pediat, Cincinnati, OH 45229 USAKonstan, Michael W.论文数: 0 引用数: 0 h-index: 0机构: Case Western Reserve Univ, Rainbow Babies & Childrens Hosp, Sch Med, Dept Pediat,Div Pulmonol, Cleveland, OH 44106 USA Cincinnati Childrens Hosp, Med Ctr, Dept Pediat, Cincinnati, OH 45229 USA
- [2] Cystic fibrosis[J]. LANCET, 2009, 373 (9678) : 1891 - 1904O'Sullivan, Brian P.论文数: 0 引用数: 0 h-index: 0机构: Univ Massachusetts, Sch Med, Dept Pediat, Worcester, MA 01655 USA Univ Massachusetts, Sch Med, Dept Pediat, Worcester, MA 01655 USAFreedman, Steven D.论文数: 0 引用数: 0 h-index: 0机构: Harvard Univ, Beth Israel Deaconess Med Ctr, Sch Med, Dept Med, Boston, MA 02215 USA Univ Massachusetts, Sch Med, Dept Pediat, Worcester, MA 01655 USA
- [3] A CFTR Potentiator in Patients with Cystic Fibrosis and the G551D Mutation[J]. NEW ENGLAND JOURNAL OF MEDICINE, 2011, 365 (18) : 1663 - 1672Ramsey, Bonnie W.论文数: 0 引用数: 0 h-index: 0机构: Univ Washington, Dept Pediat, Seattle, WA 98105 USA Seattle Childrens Hosp, Seattle, WA USA Univ Washington, Dept Pediat, Seattle, WA 98105 USADavies, Jane论文数: 0 引用数: 0 h-index: 0机构: Royal Brompton & Harefield Natl Hlth Serv Fdn Tru, Res Biomed Res Unit, London, England Univ Washington, Dept Pediat, Seattle, WA 98105 USAMcElvaney, N. Gerard论文数: 0 引用数: 0 h-index: 0机构: Beaumont Hosp, Royal Coll Surg Ireland, Beaumont, TX USA Univ Washington, Dept Pediat, Seattle, WA 98105 USATullis, Elizabeth论文数: 0 引用数: 0 h-index: 0机构: Univ Toronto, St Michaels Hosp, Toronto, ON M5B 1W8, Canada Univ Washington, Dept Pediat, Seattle, WA 98105 USABell, Scott C.论文数: 0 引用数: 0 h-index: 0机构: Prince Charles Hosp, Dept Thorac Med, Brisbane, Qld 4032, Australia Univ Queensland, Queensland Childrens Med Res Inst, Brisbane, Qld, Australia Univ Washington, Dept Pediat, Seattle, WA 98105 USADrevinek, Pavel论文数: 0 引用数: 0 h-index: 0机构: Charles Univ Prague, Dept Pediat, Sch Med 2, Prague, Czech Republic Univ Washington, Dept Pediat, Seattle, WA 98105 USAGriese, Matthias论文数: 0 引用数: 0 h-index: 0机构: Univ Munich, Dr Von Haunerschen Kinderspital, D-80337 Munich, Germany Univ Washington, Dept Pediat, Seattle, WA 98105 USAMcKone, Edward F.论文数: 0 引用数: 0 h-index: 0机构: St Vincents Univ Hosp, Dublin 4, Ireland Univ Washington, Dept Pediat, Seattle, WA 98105 USAWainwright, Claire E.论文数: 0 引用数: 0 h-index: 0机构: St Vincents Univ Hosp, Dublin 4, Ireland Univ Queensland, Queensland Childrens Med Res Inst, Brisbane, Qld, Australia Univ Washington, Dept Pediat, Seattle, WA 98105 USAKonstan, Michael W.论文数: 0 引用数: 0 h-index: 0机构: Case Western Reserve Univ, Sch Med, Rainbow Babies & Childrens Hosp, Cleveland, OH USA Univ Washington, Dept Pediat, Seattle, WA 98105 USAMoss, Richard论文数: 0 引用数: 0 h-index: 0机构: Stanford Univ, Sch Med, Stanford, CA 94305 USA Univ Washington, Dept Pediat, Seattle, WA 98105 USARatjen, Felix论文数: 0 引用数: 0 h-index: 0机构: Hosp Sick Children, Dept Pediat, Toronto, ON M5G 1X8, Canada Univ Washington, Dept Pediat, Seattle, WA 98105 USASermet-Gaudelus, Isabelle论文数: 0 引用数: 0 h-index: 0机构: Hop Necker Enfants Malad, Ctr Invest Clin, Paris, France Univ Washington, Dept Pediat, Seattle, WA 98105 USARowe, Steven M.论文数: 0 引用数: 0 h-index: 0机构: Univ Alabama Birmingham, Birmingham, AL USA Univ Washington, Dept Pediat, Seattle, WA 98105 USADong, Qunming论文数: 0 引用数: 0 h-index: 0机构: Vertex Pharmaceut, Cambridge, MA USA Univ Washington, Dept Pediat, Seattle, WA 98105 USARodriguez, Sally论文数: 0 引用数: 0 h-index: 0机构: Vertex Pharmaceut, Cambridge, MA USA Univ Washington, Dept Pediat, Seattle, WA 98105 USAYen, Karl论文数: 0 引用数: 0 h-index: 0机构: Vertex Pharmaceut, Cambridge, MA USA Univ Washington, Dept Pediat, Seattle, WA 98105 USAOrdonez, Claudia论文数: 0 引用数: 0 h-index: 0机构: Vertex Pharmaceut, Cambridge, MA USA Univ Washington, Dept Pediat, Seattle, WA 98105 USAElborn, J. Stuart论文数: 0 引用数: 0 h-index: 0机构: Queens Univ Belfast, Ctr Infect & Immun, Belfast, Antrim, North Ireland Univ Washington, Dept Pediat, Seattle, WA 98105 USA
- [4] Ataluren (PTC124) Induces Cystic Fibrosis Transmembrane Conductance Regulator Protein Expression and Activity in Children with Nonsense Mutation Cystic Fibrosis[J]. AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE, 2010, 182 (10) : 1262 - 1272Sermet-Gaudelus, Isabelle论文数: 0 引用数: 0 h-index: 0机构: Hop Necker Enfants Malad, F-75015 Paris, France Hop Necker Enfants Malad, F-75015 Paris, FranceDe Boeck, Kris论文数: 0 引用数: 0 h-index: 0机构: Univ Leuven, Louvain, Belgium Hop Necker Enfants Malad, F-75015 Paris, FranceCasimir, Georges J.论文数: 0 引用数: 0 h-index: 0机构: Hop Univ Enfants Reine Fabiola, Brussels, Belgium Hop Necker Enfants Malad, F-75015 Paris, FranceVermeulen, Francois论文数: 0 引用数: 0 h-index: 0机构: Univ Leuven, Louvain, Belgium Hop Necker Enfants Malad, F-75015 Paris, FranceLeal, Teresinha论文数: 0 引用数: 0 h-index: 0机构: Catholic Univ Louvain, B-1200 Brussels, Belgium Hop Necker Enfants Malad, F-75015 Paris, FranceMogenet, Agnes论文数: 0 引用数: 0 h-index: 0机构: Hop Necker Enfants Malad, F-75015 Paris, France Hop Necker Enfants Malad, F-75015 Paris, FranceRoussel, Delphine论文数: 0 引用数: 0 h-index: 0机构: Hop Necker Enfants Malad, F-75015 Paris, France Hop Necker Enfants Malad, F-75015 Paris, FranceFritsch, Janine论文数: 0 引用数: 0 h-index: 0机构: Hop Necker Enfants Malad, F-75015 Paris, France Hop Necker Enfants Malad, F-75015 Paris, FranceHanssens, Laurence论文数: 0 引用数: 0 h-index: 0机构: Hop Univ Enfants Reine Fabiola, Brussels, Belgium Hop Necker Enfants Malad, F-75015 Paris, FranceHirawat, Samit论文数: 0 引用数: 0 h-index: 0机构: PTC Therapeut, S Plainfield, NJ USA Hop Necker Enfants Malad, F-75015 Paris, FranceMiller, Nilsen L.论文数: 0 引用数: 0 h-index: 0机构: PTC Therapeut, S Plainfield, NJ USA Hop Necker Enfants Malad, F-75015 Paris, FranceConstantine, Scott论文数: 0 引用数: 0 h-index: 0机构: PTC Therapeut, S Plainfield, NJ USA Hop Necker Enfants Malad, F-75015 Paris, FranceReha, Allen论文数: 0 引用数: 0 h-index: 0机构: PTC Therapeut, S Plainfield, NJ USA Hop Necker Enfants Malad, F-75015 Paris, FranceAjayi, Temitayo论文数: 0 引用数: 0 h-index: 0机构: PTC Therapeut, S Plainfield, NJ USA Hop Necker Enfants Malad, F-75015 Paris, FranceElfring, Gary L.论文数: 0 引用数: 0 h-index: 0机构: PTC Therapeut, S Plainfield, NJ USA Hop Necker Enfants Malad, F-75015 Paris, FranceMiller, Langdon L.论文数: 0 引用数: 0 h-index: 0机构: PTC Therapeut, S Plainfield, NJ USA Hop Necker Enfants Malad, F-75015 Paris, France
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- [7] Ivacaftor potentiation of multiple CFTR channels with gating mutations[J]. JOURNAL OF CYSTIC FIBROSIS, 2012, 11 (03) : 237 - 245Yu, Haihui论文数: 0 引用数: 0 h-index: 0机构: Vertex Pharmaceut Inc, Cambridge, MA 02139 USA Vertex Pharmaceut Inc, Cambridge, MA 02139 USABurton, Bill论文数: 0 引用数: 0 h-index: 0机构: Vertex Pharmaceut Inc, Cambridge, MA 02139 USA Vertex Pharmaceut Inc, Cambridge, MA 02139 USAHuang, Chien-Jung论文数: 0 引用数: 0 h-index: 0机构: Vertex Pharmaceut Inc, Cambridge, MA 02139 USA Vertex Pharmaceut Inc, Cambridge, MA 02139 USAWorley, Jennings论文数: 0 引用数: 0 h-index: 0机构: Vertex Pharmaceut Inc, Cambridge, MA 02139 USA Vertex Pharmaceut Inc, Cambridge, MA 02139 USACao, Dong论文数: 0 引用数: 0 h-index: 0机构: Vertex Pharmaceut Inc, Cambridge, MA 02139 USA Vertex Pharmaceut Inc, Cambridge, MA 02139 USAJohnson, James P., Jr.论文数: 0 引用数: 0 h-index: 0机构: Vertex Pharmaceut Inc, Cambridge, MA 02139 USA Vertex Pharmaceut Inc, Cambridge, MA 02139 USAUrrutia, Art论文数: 0 引用数: 0 h-index: 0机构: Vertex Pharmaceut Inc, Cambridge, MA 02139 USA Vertex Pharmaceut Inc, Cambridge, MA 02139 USAJoubran, John论文数: 0 引用数: 0 h-index: 0机构: Vertex Pharmaceut Inc, Cambridge, MA 02139 USA Vertex Pharmaceut Inc, Cambridge, MA 02139 USASeepersaud, Sheila论文数: 0 引用数: 0 h-index: 0机构: Vertex Pharmaceut Inc, Cambridge, MA 02139 USA Vertex Pharmaceut Inc, Cambridge, MA 02139 USASussky, Katherine论文数: 0 引用数: 0 h-index: 0机构: Vertex Pharmaceut Inc, Cambridge, MA 02139 USA Vertex Pharmaceut Inc, Cambridge, MA 02139 USAHoffman, Beth J.论文数: 0 引用数: 0 h-index: 0机构: Vertex Pharmaceut Inc, Cambridge, MA 02139 USA Vertex Pharmaceut Inc, Cambridge, MA 02139 USAVan Goor, Fredrick论文数: 0 引用数: 0 h-index: 0机构: Vertex Pharmaceut Inc, Cambridge, MA 02139 USA Vertex Pharmaceut Inc, Cambridge, MA 02139 USA