共 50 条
- [1] Phase 2a Study of Ataluren-Mediated Dystrophin Production in Patients with Nonsense Mutation Duchenne Muscular DystrophyPLOS ONE, 2013, 8 (12):Finkel, Richard S.论文数: 0 引用数: 0 h-index: 0机构: Childrens Hosp Philadelphia, Philadelphia, PA 19104 USA Nemours Childrens Hosp, Orlando, FL USA Childrens Hosp Philadelphia, Philadelphia, PA 19104 USAFlanigan, Kevin M.论文数: 0 引用数: 0 h-index: 0机构: Univ Utah Sch Med, Salt Lake City, UT USA Childrens Hosp Philadelphia, Philadelphia, PA 19104 USAWong, Brenda论文数: 0 引用数: 0 h-index: 0机构: Cincinnati Childrens Hosp Med Ctr, Cincinnati, OH 45229 USA Childrens Hosp Philadelphia, Philadelphia, PA 19104 USABoennemann, Carsten论文数: 0 引用数: 0 h-index: 0机构: Childrens Hosp Philadelphia, Philadelphia, PA 19104 USA Childrens Hosp Philadelphia, Philadelphia, PA 19104 USASampson, Jacinda论文数: 0 引用数: 0 h-index: 0机构: Univ Utah Sch Med, Salt Lake City, UT USA Childrens Hosp Philadelphia, Philadelphia, PA 19104 USASweeney, H. Lee论文数: 0 引用数: 0 h-index: 0机构: Univ Penn, Perelman Sch Med, Philadelphia, PA 19104 USA Childrens Hosp Philadelphia, Philadelphia, PA 19104 USAReha, Allen论文数: 0 引用数: 0 h-index: 0机构: PTC Therapeut Inc, South Plainfield, NJ USA Childrens Hosp Philadelphia, Philadelphia, PA 19104 USANorthcutt, Valerie J.论文数: 0 引用数: 0 h-index: 0机构: PTC Therapeut Inc, South Plainfield, NJ USA Childrens Hosp Philadelphia, Philadelphia, PA 19104 USAElfring, Gary论文数: 0 引用数: 0 h-index: 0机构: PTC Therapeut Inc, South Plainfield, NJ USA Childrens Hosp Philadelphia, Philadelphia, PA 19104 USABarth, Jay论文数: 0 引用数: 0 h-index: 0机构: PTC Therapeut Inc, South Plainfield, NJ USA Childrens Hosp Philadelphia, Philadelphia, PA 19104 USAPeltz, Stuart W.论文数: 0 引用数: 0 h-index: 0机构: PTC Therapeut Inc, South Plainfield, NJ USA Childrens Hosp Philadelphia, Philadelphia, PA 19104 USA
- [2] Duchenne Muscular Dystrophy: Ataluren in Nonsense MutationFORTSCHRITTE DER NEUROLOGIE PSYCHIATRIE, 2018, 86 (04)Manych, Matthias论文数: 0 引用数: 0 h-index: 0
- [3] Demographics of patients with nonsense mutation Duchenne muscular dystrophy receiving ataluren in the STRIDE RegistryNEUROMUSCULAR DISORDERS, 2020, 30 : S62 - S62Muntoni, F.论文数: 0 引用数: 0 h-index: 0机构: UCL, London, England UCL, London, EnglandMercuri, E.论文数: 0 引用数: 0 h-index: 0机构: Catholic Univ, Rome, Italy UCL, London, EnglandBuccella, F.论文数: 0 引用数: 0 h-index: 0机构: Parent Project Italy APS, Rome, Italy UCL, London, EnglandDesguerre, I.论文数: 0 引用数: 0 h-index: 0机构: Paris V Descartes Univ, Paris, France UCL, London, EnglandKirschner, J.论文数: 0 引用数: 0 h-index: 0机构: Univ Hosp Bonn, Bonn, Germany UCL, London, EnglandNascimento Osorio, A.论文数: 0 引用数: 0 h-index: 0机构: Univ Barcelona, Barcelona, Spain UCL, London, EnglandTulinius, M.论文数: 0 引用数: 0 h-index: 0机构: Gothenburg Univ, Gothenburg, Sweden UCL, London, EnglandJiang, J.论文数: 0 引用数: 0 h-index: 0机构: PTC Therapeut, South Plainfield, NJ USA UCL, London, EnglandKristensen, A.论文数: 0 引用数: 0 h-index: 0机构: PTC Therapeut, South Plainfield, NJ USA UCL, London, EnglandJohnson, S.论文数: 0 引用数: 0 h-index: 0机构: PTC Therapeut, South Plainfield, NJ USA UCL, London, EnglandAble, R.论文数: 0 引用数: 0 h-index: 0机构: PTC Therapeut, South Plainfield, NJ USA UCL, London, EnglandTrifillis, P.论文数: 0 引用数: 0 h-index: 0机构: PTC Therapeut, South Plainfield, NJ USA UCL, London, EnglandSantos, C.论文数: 0 引用数: 0 h-index: 0机构: PTC Therapeut, South Plainfield, NJ USA UCL, London, England
- [4] Safety and Tolerability of Ataluren in a Phase 3 Study of Patients with Nonsense Mutation Duchenne Muscular DystrophyNEUROLOGY, 2016, 86Campbell, Craig论文数: 0 引用数: 0 h-index: 0机构: London Hlth Sci Ctr, London, ON, Canada Western Univ, London, ON, Canada Cincinnati Childrens Hosp Med Ctr, Cincinnati, OH 45229 USAShieh, Perry论文数: 0 引用数: 0 h-index: 0机构: Univ Calif Los Angeles, Los Angeles, CA USA Cincinnati Childrens Hosp Med Ctr, Cincinnati, OH 45229 USASejersen, Thomas论文数: 0 引用数: 0 h-index: 0机构: Karolinska Inst, Stockholm, Sweden Cincinnati Childrens Hosp Med Ctr, Cincinnati, OH 45229 USALuo, Xiaohui论文数: 0 引用数: 0 h-index: 0机构: PTC Therapeut, South Plainfield, NJ USA Cincinnati Childrens Hosp Med Ctr, Cincinnati, OH 45229 USAElfring, Gary论文数: 0 引用数: 0 h-index: 0机构: PTC Therapeut, South Plainfield, NJ USA Cincinnati Childrens Hosp Med Ctr, Cincinnati, OH 45229 USAKroger, Hans论文数: 0 引用数: 0 h-index: 0机构: PTC Therapeut, South Plainfield, NJ USA Cincinnati Childrens Hosp Med Ctr, Cincinnati, OH 45229 USARiebling, Peter论文数: 0 引用数: 0 h-index: 0机构: PTC Therapeut, South Plainfield, NJ USA Cincinnati Childrens Hosp Med Ctr, Cincinnati, OH 45229 USAOng, Tuyen论文数: 0 引用数: 0 h-index: 0机构: PTC Therapeut, South Plainfield, NJ USA Cincinnati Childrens Hosp Med Ctr, Cincinnati, OH 45229 USASpiegel, Robert论文数: 0 引用数: 0 h-index: 0机构: PTC Therapeut, South Plainfield, NJ USA Cincinnati Childrens Hosp Med Ctr, Cincinnati, OH 45229 USAPeltz, Stuart论文数: 0 引用数: 0 h-index: 0机构: PTC Therapeut, South Plainfield, NJ USA Cincinnati Childrens Hosp Med Ctr, Cincinnati, OH 45229 USAWong, Brenda论文数: 0 引用数: 0 h-index: 0机构: Cincinnati Childrens Hosp Med Ctr, Cincinnati, OH 45229 USA Cincinnati Childrens Hosp Med Ctr, Cincinnati, OH 45229 USA
- [5] Meta-analyses of ataluren in patients with nonsense mutation Duchenne muscular dystrophyNEUROMUSCULAR DISORDERS, 2018, 28 : S12 - S12Muntoni, F.论文数: 0 引用数: 0 h-index: 0机构: UCL, London, England UCL, London, EnglandMercuri, E.论文数: 0 引用数: 0 h-index: 0机构: Catholic Univ, Rome, Italy UCL, London, EnglandLuo, X.论文数: 0 引用数: 0 h-index: 0机构: PTC Therapeut Inc, South Plainfield, NJ USA UCL, London, EnglandElfring, G.论文数: 0 引用数: 0 h-index: 0机构: PTC Therapeut Inc, South Plainfield, NJ USA UCL, London, EnglandWerner, C.论文数: 0 引用数: 0 h-index: 0机构: PTC Therapeut Germany GmbH, Frankfurt, Germany UCL, London, EnglandTrifillis, R.论文数: 0 引用数: 0 h-index: 0机构: PTC Therapeut Inc, South Plainfield, NJ USA UCL, London, EnglandPeltz, S. W.论文数: 0 引用数: 0 h-index: 0机构: PTC Therapeut Inc, South Plainfield, NJ USA UCL, London, EnglandMcDonald, C. M.论文数: 0 引用数: 0 h-index: 0机构: Univ Calif Davis, Sch Med, Davis, CA 95616 USA UCL, London, England
- [6] Meta-Analyses of Ataluren in Patients with Nonsense Mutation Duchenne Muscular DystrophyANNALS OF NEUROLOGY, 2016, 80 : S365 - S365Campbell, C.论文数: 0 引用数: 0 h-index: 0Muntoni, F.论文数: 0 引用数: 0 h-index: 0Mercuri, E.论文数: 0 引用数: 0 h-index: 0Luo, X.论文数: 0 引用数: 0 h-index: 0Elfring, G.论文数: 0 引用数: 0 h-index: 0Kroger, H.论文数: 0 引用数: 0 h-index: 0Riebling, P.论文数: 0 引用数: 0 h-index: 0Ong, T.论文数: 0 引用数: 0 h-index: 0Spiegel, R.论文数: 0 引用数: 0 h-index: 0Peltz, S.论文数: 0 引用数: 0 h-index: 0McDonald, C.论文数: 0 引用数: 0 h-index: 0
- [7] Meta-analyses of ataluren randomized controlled trials in nonsense mutation Duchenne muscular dystrophyJOURNAL OF COMPARATIVE EFFECTIVENESS RESEARCH, 2020, 9 (14) : 973 - 984Campbell, Craig论文数: 0 引用数: 0 h-index: 0机构: Western Univ, Schulich Sch Med & Dent, London, ON N6A 5C1, Canada Western Univ, Schulich Sch Med & Dent, London, ON N6A 5C1, CanadaBarohn, Richard J.论文数: 0 引用数: 0 h-index: 0机构: Univ Kansas, Med Ctr, Kansas City, KS 66160 USA Western Univ, Schulich Sch Med & Dent, London, ON N6A 5C1, CanadaBertini, Enrico论文数: 0 引用数: 0 h-index: 0机构: Bambino Gesu Childrens Res Hosp, I-00146 Rome, Italy Western Univ, Schulich Sch Med & Dent, London, ON N6A 5C1, CanadaChabrol, Brigitte论文数: 0 引用数: 0 h-index: 0机构: Hop La Timone, Unite Med Infantile, F-13385 Marseille, France Western Univ, Schulich Sch Med & Dent, London, ON N6A 5C1, CanadaComi, Giacomo Pietro论文数: 0 引用数: 0 h-index: 0机构: Univ Milan, IRCCS Fdn Ca Granda Osped Maggiore Policlin, Dino Ferrari Ctr, Dept Pathophysiol & Transplantat, I-20122 Milan, Italy Western Univ, Schulich Sch Med & Dent, London, ON N6A 5C1, CanadaDarras, Basil T.论文数: 0 引用数: 0 h-index: 0机构: Harvard Med Sch, Boston Childrens Hosp, Boston, MA 02115 USA Western Univ, Schulich Sch Med & Dent, London, ON N6A 5C1, CanadaFinkel, Richard S.论文数: 0 引用数: 0 h-index: 0机构: Childrens Hosp Philadelphia, Philadelphia, PA 19104 USA St Jude Childrens Res Hosp, Memphis, TN 38105 USA Western Univ, Schulich Sch Med & Dent, London, ON N6A 5C1, CanadaFlanigan, Kevin M.论文数: 0 引用数: 0 h-index: 0机构: Nationwide Childrens Hosp, Columbus, OH 43205 USA Western Univ, Schulich Sch Med & Dent, London, ON N6A 5C1, CanadaGoemans, Nathalie论文数: 0 引用数: 0 h-index: 0机构: Katholieke Univ Leuven, Univ Hosp Leuven, B-3000 Leuven, Belgium Western Univ, Schulich Sch Med & Dent, London, ON N6A 5C1, CanadaIannaccone, Susan T.论文数: 0 引用数: 0 h-index: 0机构: Univ Texas Southwestern Med Ctr Dallas, Dallas, TX 75390 USA Western Univ, Schulich Sch Med & Dent, London, ON N6A 5C1, CanadaJones, Kristi J.论文数: 0 引用数: 0 h-index: 0机构: Childrens Hosp Westmead, Kids Neurosci, Westmead, NSW 2145, Australia Western Univ, Schulich Sch Med & Dent, London, ON N6A 5C1, CanadaKirschner, Janbernd论文数: 0 引用数: 0 h-index: 0机构: Univ Freiburg, Med Ctr, Dept Neuropediat & Muscle Disorders, D-79110 Freiburg, Germany Western Univ, Schulich Sch Med & Dent, London, ON N6A 5C1, CanadaMah, Jean K.论文数: 0 引用数: 0 h-index: 0机构: Univ Calgary, Alberta Childrens Hosp, Dept Pediat, Div Pediat Neurol, Calgary, AB T3B 6A8, Canada Western Univ, Schulich Sch Med & Dent, London, ON N6A 5C1, CanadaMathews, Katherine D.论文数: 0 引用数: 0 h-index: 0机构: Univ Iowa, Dept Pediat, Div Pediat Neurol, Carver Coll Med, Iowa City, IA 52242 USA Univ Iowa, Dept Neurol, Div Pediat Neurol, Carver Coll Med, Iowa City, IA 52242 USA Western Univ, Schulich Sch Med & Dent, London, ON N6A 5C1, CanadaMcDonald, Craig M.论文数: 0 引用数: 0 h-index: 0机构: Univ Calif Davis Hlth, Sacramento, CA 95817 USA Western Univ, Schulich Sch Med & Dent, London, ON N6A 5C1, CanadaMercuri, Eugenio论文数: 0 引用数: 0 h-index: 0机构: Catholic Univ, Dept Pediat Neurol, I-00168 Rome, Italy Western Univ, Schulich Sch Med & Dent, London, ON N6A 5C1, CanadaNevo, Yoram论文数: 0 引用数: 0 h-index: 0机构: Tel Aviv Univ, Schneider Childrens Med Ctr, IL-6997801 Tel Aviv, Israel Western Univ, Schulich Sch Med & Dent, London, ON N6A 5C1, CanadaPereon, Yann论文数: 0 引用数: 0 h-index: 0机构: Hop Hotel Dieu, Reference Ctr Neuromuscular Disorders AOC, F-44000 Nantes, France Western Univ, Schulich Sch Med & Dent, London, ON N6A 5C1, CanadaBen Renfroe, J.论文数: 0 引用数: 0 h-index: 0机构: Child Neurol Ctr Northwest Florida, Gulf Breeze, FL 32561 USA Western Univ, Schulich Sch Med & Dent, London, ON N6A 5C1, CanadaRyan, Monique M.论文数: 0 引用数: 0 h-index: 0机构: Royal Childrens Hosp, Parkville, Vic 3052, Australia Western Univ, Schulich Sch Med & Dent, London, ON N6A 5C1, CanadaSampson, Jacinda B.论文数: 0 引用数: 0 h-index: 0机构: Stanford Univ, Dept Neurol & Neurol Sci, Med Ctr, Stanford, CA 94305 USA Western Univ, Schulich Sch Med & Dent, London, ON N6A 5C1, Canada论文数: 引用数: h-index:机构:Sejersen, Thomas论文数: 0 引用数: 0 h-index: 0机构: Karolinska Inst, Karolinska Univ Hosp, S-17176 Stockholm, Sweden Western Univ, Schulich Sch Med & Dent, London, ON N6A 5C1, CanadaSelby, Kathryn论文数: 0 引用数: 0 h-index: 0机构: British Columbia Childrens Hosp, Div Neurol, Vancouver, BC V6H 3N1, Canada Western Univ, Schulich Sch Med & Dent, London, ON N6A 5C1, CanadaTulinius, Mar论文数: 0 引用数: 0 h-index: 0机构: Gothenburg Univ, Queen Silvia Childrens Hosp, S-41685 Gothenburg, Sweden Western Univ, Schulich Sch Med & Dent, London, ON N6A 5C1, CanadaVilchez, Juan J.论文数: 0 引用数: 0 h-index: 0机构: Hosp Univ & Politecn La Fe, CIBERER, Valencia 46026, Spain Western Univ, Schulich Sch Med & Dent, London, ON N6A 5C1, CanadaVoit, Thomas论文数: 0 引用数: 0 h-index: 0机构: UCL, NIHR Great Ormond St Hosp Biomed Res Ctr, London WC1N 1EH, England Great Ormond St Hosp Trust, UCL Great Ormond St Inst Child Hlth, London WC1N 1EH, England Western Univ, Schulich Sch Med & Dent, London, ON N6A 5C1, CanadaWei, Lee-Jen论文数: 0 引用数: 0 h-index: 0机构: Harvard Univ, Harvard TH Chan Sch Publ Hlth, Boston, MA 02115 USA Western Univ, Schulich Sch Med & Dent, London, ON N6A 5C1, CanadaWong, Brenda L.论文数: 0 引用数: 0 h-index: 0机构: Univ Massachusetts, UMass, Med Sch, Worcester, MA 01655 USA Western Univ, Schulich Sch Med & Dent, London, ON N6A 5C1, CanadaElfring, Gary论文数: 0 引用数: 0 h-index: 0机构: PTC Therapeut Inc, South Plainfield, NJ 07080 USA Western Univ, Schulich Sch Med & Dent, London, ON N6A 5C1, CanadaSouza, Marcio论文数: 0 引用数: 0 h-index: 0机构: PTC Therapeut Inc, South Plainfield, NJ 07080 USA Western Univ, Schulich Sch Med & Dent, London, ON N6A 5C1, CanadaMcIntosh, Joseph论文数: 0 引用数: 0 h-index: 0机构: PTC Therapeut Inc, South Plainfield, NJ 07080 USA Western Univ, Schulich Sch Med & Dent, London, ON N6A 5C1, CanadaTrifillis, Panayiota论文数: 0 引用数: 0 h-index: 0机构: PTC Therapeut Inc, South Plainfield, NJ 07080 USA Western Univ, Schulich Sch Med & Dent, London, ON N6A 5C1, CanadaPeltz, Stuart W.论文数: 0 引用数: 0 h-index: 0机构: PTC Therapeut Inc, South Plainfield, NJ 07080 USA Western Univ, Schulich Sch Med & Dent, London, ON N6A 5C1, CanadaMuntoni, Francesco论文数: 0 引用数: 0 h-index: 0机构: UCL, NIHR Great Ormond St Hosp Biomed Res Ctr, London WC1N 1EH, England Great Ormond St Hosp Trust, UCL Great Ormond St Inst Child Hlth, London WC1N 1EH, England Western Univ, Schulich Sch Med & Dent, London, ON N6A 5C1, Canada
- [8] META-ANALYSES OF ATALUREN IN PATIENTS WITH NONSENSE MUTATION DUCHENNE MUSCULAR DYSTROPHY (NMDMD)MUSCLE & NERVE, 2016, 54 : 12 - 13Butterfield, Russell J.论文数: 0 引用数: 0 h-index: 0Luo, Xiaohui论文数: 0 引用数: 0 h-index: 0Elfring, Gary论文数: 0 引用数: 0 h-index: 0Kroger, Hans论文数: 0 引用数: 0 h-index: 0Riebling, Peter论文数: 0 引用数: 0 h-index: 0McIntosh, Joseph论文数: 0 引用数: 0 h-index: 0Ong, Tuyen论文数: 0 引用数: 0 h-index: 0Spiegel, Robert论文数: 0 引用数: 0 h-index: 0Peltz, Stuart W.论文数: 0 引用数: 0 h-index: 0McDonald, Craig论文数: 0 引用数: 0 h-index: 0
- [9] Ataluren for treating Duchenne muscular dystrophy with a nonsense mutation in the dystrophin gene - evaluation through a managed access agreementNEUROMUSCULAR DISORDERS, 2019, 29 : S167 - S167Ayyar-Gupta, V.论文数: 0 引用数: 0 h-index: 0机构: UCL, London, England UCL, London, EnglandRidout, D.论文数: 0 引用数: 0 h-index: 0机构: UCL, London, England UCL, London, EnglandManzur, A.论文数: 0 引用数: 0 h-index: 0机构: Great Ormond St Hosp Sick Children, London, England UCL, London, EnglandMuntoni, F.论文数: 0 引用数: 0 h-index: 0机构: UCL, London, England UCL, London, England
- [10] Updated demographics and safety data from patients with nonsense mutation Duchenne muscular dystrophy receiving ataluren in the STRIDE RegistryNEUROMUSCULAR DISORDERS, 2021, 31 : S91 - S92Muntoni, F.论文数: 0 引用数: 0 h-index: 0机构: UCL, London, England UCL, London, EnglandBuccella, F.论文数: 0 引用数: 0 h-index: 0机构: Duchenne Parent Project Italy, Rome, Italy UCL, London, EnglandDesguerre, I.论文数: 0 引用数: 0 h-index: 0机构: Hop Necker Enfants Malad, Paris, France UCL, London, EnglandKirschner, J.论文数: 0 引用数: 0 h-index: 0机构: Univ Freiburg, Med Ctr, Freiburg, Germany UCL, London, EnglandMercuri, E.论文数: 0 引用数: 0 h-index: 0机构: Catholic Univ, Rome, Italy UCL, London, EnglandOsorio, A. Nascimento论文数: 0 引用数: 0 h-index: 0机构: Hosp St Joan de Deu, Barcelona, Spain UCL, London, EnglandTulinius, M.论文数: 0 引用数: 0 h-index: 0机构: Gothenburg Univ, Gothenburg, Sweden UCL, London, EnglandJohnson, S.论文数: 0 引用数: 0 h-index: 0机构: PTC Therapeut Inc, South Plainfield, NJ USA UCL, London, EnglandWerner, C.论文数: 0 引用数: 0 h-index: 0机构: PTC Therapeut Germany GmbH, Frankfurt, Germany UCL, London, EnglandKristensen, A.论文数: 0 引用数: 0 h-index: 0机构: PTC Therapeut Inc, South Plainfield, NJ USA UCL, London, EnglandJiang, J.论文数: 0 引用数: 0 h-index: 0机构: PTC Therapeut Inc, South Plainfield, NJ USA UCL, London, EnglandLi, J.论文数: 0 引用数: 0 h-index: 0机构: PTC Therapeut Inc, South Plainfield, NJ USA UCL, London, EnglandTrifillis, P.论文数: 0 引用数: 0 h-index: 0机构: PTC Therapeut Inc, South Plainfield, NJ USA UCL, London, EnglandSantos, C.论文数: 0 引用数: 0 h-index: 0机构: PTC Therapeut Inc, South Plainfield, NJ USA UCL, London, England