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- [31] RAINBOWFISH: A study of risdiplam in infants with presymptomatic spinal muscular atrophy (SMA)NEUROMUSCULAR DISORDERS, 2021, 31 : S48 - S48论文数: 引用数: h-index:机构:Al-Muhaizea, M.论文数: 0 引用数: 0 h-index: 0机构: King Faisal Specialist Hosp & Res Ctr, Riyadh, Saudi Arabia Univ Oxford, Oxford, EnglandFarrar, M.论文数: 0 引用数: 0 h-index: 0机构: UNSW, Sydney, NSW, Australia Univ Oxford, Oxford, EnglandNelson, L.论文数: 0 引用数: 0 h-index: 0机构: UT Southwestern Med Ctr, Dallas, TX USA Univ Oxford, Oxford, EnglandPrufer, A.论文数: 0 引用数: 0 h-index: 0机构: Fed Uni Rio de Janeiro, Rio De Janeiro, Brazil Univ Oxford, Oxford, EnglandFinkel, R.论文数: 0 引用数: 0 h-index: 0机构: St Jude Childrens Res Hosp, Memphis, TN USA Univ Oxford, Oxford, EnglandWang, Y.论文数: 0 引用数: 0 h-index: 0机构: Fudan Univ, Childrens Hosp, Shanghai, Peoples R China Univ Oxford, Oxford, EnglandZanoteli, E.论文数: 0 引用数: 0 h-index: 0机构: Univ Sao Paulo FMUSP, Sao Paulo, Brazil Univ Oxford, Oxford, EnglandEl-Khairi, M.论文数: 0 引用数: 0 h-index: 0机构: Roche Prod Ltd, Welwyn Garden City, Herts, England Univ Oxford, Oxford, EnglandGerber, M.论文数: 0 引用数: 0 h-index: 0机构: F Hoffmann La Roche Ltd, Basel, Switzerland Univ Oxford, Oxford, EnglandGorni, K.论文数: 0 引用数: 0 h-index: 0机构: F Hoffmann La Roche Ltd, Basel, Switzerland Univ Oxford, Oxford, EnglandKletzl, H.论文数: 0 引用数: 0 h-index: 0机构: Roche Innovat Ctr Basel, Basel, Switzerland Univ Oxford, Oxford, EnglandPalfreeman, L.论文数: 0 引用数: 0 h-index: 0机构: Roche Prod Ltd, Welwyn Garden City, Herts, England Univ Oxford, Oxford, EnglandScalco, R.论文数: 0 引用数: 0 h-index: 0机构: F Hoffmann La Roche Ltd, Basel, Switzerland Univ Oxford, Oxford, EnglandBertini, E.论文数: 0 引用数: 0 h-index: 0机构: Childrens Res Hosp IRCCS, Rome, Italy Univ Oxford, Oxford, England
- [32] RAINBOWFISH: A Study of Risdiplam in Newborns with Presymptomatic Spinal Muscular Atrophy (SMA)NEUROLOGY, 2021, 96 (15)Finkel, Richard S.论文数: 0 引用数: 0 h-index: 0机构: St Jude Childrens Res Hosp, Ctr Expt Neurotherapeut, 332 N Lauderdale St, Memphis, TN 38105 USA St Jude Childrens Res Hosp, Ctr Expt Neurotherapeut, 332 N Lauderdale St, Memphis, TN 38105 USAAl-Muhaizea, Mohammad论文数: 0 引用数: 0 h-index: 0机构: King Faisal Specialist Hosp & Res Ctr, Dept Neurosci, Riyadh, Saudi Arabia St Jude Childrens Res Hosp, Ctr Expt Neurotherapeut, 332 N Lauderdale St, Memphis, TN 38105 USAFarrar, Michelle A.论文数: 0 引用数: 0 h-index: 0机构: UNSW Sydney, Sydney Childrens Hosp Network, Sydney, NSW, Australia UNSW Sydney, UNSW Med, Sydney, NSW, Australia St Jude Childrens Res Hosp, Ctr Expt Neurotherapeut, 332 N Lauderdale St, Memphis, TN 38105 USANelson, Leslie论文数: 0 引用数: 0 h-index: 0机构: Univ Texas Southwestern Med Ctr Dallas, Dallas, TX 75390 USA St Jude Childrens Res Hosp, Ctr Expt Neurotherapeut, 332 N Lauderdale St, Memphis, TN 38105 USAPrufer, Alexandra论文数: 0 引用数: 0 h-index: 0机构: Fed Uni Rio De Janeiro, Rio De Janeiro, Brazil St Jude Childrens Res Hosp, Ctr Expt Neurotherapeut, 332 N Lauderdale St, Memphis, TN 38105 USAServais, Laurent论文数: 0 引用数: 0 h-index: 0机构: Univ Oxford, Dept Paediat, MDUK Oxford Neuromuscular Ctr, Oxford, England Univ Hosp Liege, Div Child Neurol, Dept Pediat, Ctr References Malad Neuromusculaires, Liege, Belgium Univ Liege, Liege, Belgium Hop Armand Trousseau, I Mot, Paris, France St Jude Childrens Res Hosp, Ctr Expt Neurotherapeut, 332 N Lauderdale St, Memphis, TN 38105 USAWang, Yi论文数: 0 引用数: 0 h-index: 0机构: Fudan Univ, Childrens Hosp, Shanghai, Peoples R China St Jude Childrens Res Hosp, Ctr Expt Neurotherapeut, 332 N Lauderdale St, Memphis, TN 38105 USAZanoteli, Edmar论文数: 0 引用数: 0 h-index: 0机构: Univ Sao Paulo, Fac Med, Dept Neurol, Sao Paulo, Brazil St Jude Childrens Res Hosp, Ctr Expt Neurotherapeut, 332 N Lauderdale St, Memphis, TN 38105 USAPalfreeman, Laura论文数: 0 引用数: 0 h-index: 0机构: Roche Prod Ltd, Basel, Switzerland St Jude Childrens Res Hosp, Ctr Expt Neurotherapeut, 332 N Lauderdale St, Memphis, TN 38105 USAEl-Khairi, Muna论文数: 0 引用数: 0 h-index: 0机构: Roche Prod Ltd, Basel, Switzerland St Jude Childrens Res Hosp, Ctr Expt Neurotherapeut, 332 N Lauderdale St, Memphis, TN 38105 USAGorni, Ksenija论文数: 0 引用数: 0 h-index: 0机构: F Hoffmann La Roche Ltd, PDMA Neurosci & Rare Dis, Basel, Switzerland St Jude Childrens Res Hosp, Ctr Expt Neurotherapeut, 332 N Lauderdale St, Memphis, TN 38105 USAKletzl, Heidemarie论文数: 0 引用数: 0 h-index: 0机构: Roche Innovat Ctr Basel, Roche Pharmaceut Res & Early Dev, Basel, Switzerland St Jude Childrens Res Hosp, Ctr Expt Neurotherapeut, 332 N Lauderdale St, Memphis, TN 38105 USAGerber, Marianne论文数: 0 引用数: 0 h-index: 0机构: F Hoffmann La Roche Ltd, Pharma Dev, Safety, Basel, Switzerland St Jude Childrens Res Hosp, Ctr Expt Neurotherapeut, 332 N Lauderdale St, Memphis, TN 38105 USAScalco, Renata S.论文数: 0 引用数: 0 h-index: 0机构: F Hoffmann La Roche Ltd, Pharma Dev Neurol, Basel, Switzerland St Jude Childrens Res Hosp, Ctr Expt Neurotherapeut, 332 N Lauderdale St, Memphis, TN 38105 USABertini, Enrico论文数: 0 引用数: 0 h-index: 0机构: Bambino Gesu Childrens Res Hosp IRCCS, Dept Neurosci & Neurorehabil, Rome, Italy St Jude Childrens Res Hosp, Ctr Expt Neurotherapeut, 332 N Lauderdale St, Memphis, TN 38105 USA
- [33] FIREFISH Parts 1 and 2: 36-month safety and efficacy of risdiplam in Type 1 spinal muscular atrophyEUROPEAN JOURNAL OF NEUROLOGY, 2022, 29 : 279 - 279Deconinck, N.论文数: 0 引用数: 0 h-index: 0机构: UZ Gent, Neuromuscular Reference Ctr, Ghent, Belgium UZ Gent, Neuromuscular Reference Ctr, Ghent, BelgiumBaranello, G.论文数: 0 引用数: 0 h-index: 0机构: UCL, Great Ormond St Hosp Biomed Res Ctr, Great Ormond St Inst Child Hlth, Dubowitz Neuromuscular Ctr NIHR, London, England Great Ormond St Hosp Trust, London, England UZ Gent, Neuromuscular Reference Ctr, Ghent, BelgiumBoespflug-Tanguy, O.论文数: 0 引用数: 0 h-index: 0机构: I Mot Hop Armand Trousseau, Paris, France UZ Gent, Neuromuscular Reference Ctr, Ghent, BelgiumDay, J.论文数: 0 引用数: 0 h-index: 0机构: Stanford Univ, Dept Neurol, Palo Alto, CA USA UZ Gent, Neuromuscular Reference Ctr, Ghent, BelgiumKlein, A.论文数: 0 引用数: 0 h-index: 0机构: Univ Childrens Hosp Basel, Paediat Neurol, Basel, Switzerland UZ Gent, Neuromuscular Reference Ctr, Ghent, BelgiumMasson, R.论文数: 0 引用数: 0 h-index: 0机构: Fdn IRCCS Ist Neurol Carlo Besta, Dev Neurol Unit, Milan, Italy UZ Gent, Neuromuscular Reference Ctr, Ghent, BelgiumMazurkiewicz-Beldzinska, M.论文数: 0 引用数: 0 h-index: 0机构: Med Univ Gdansk, Dept Dev Neurol, Gdansk, Poland UZ Gent, Neuromuscular Reference Ctr, Ghent, BelgiumMercuri, E.论文数: 0 引用数: 0 h-index: 0机构: Catholic Univ, Pediat Neurol Inst, Rome, Italy Fdn Policlin Gemelli IRCCS, Nemo Pediat, Rome, Italy UZ Gent, Neuromuscular Reference Ctr, Ghent, BelgiumRose, K.论文数: 0 引用数: 0 h-index: 0机构: Univ Sydney, Fac Med & Hlth, Discipline Physiotherapy, Sydney, NSW, Australia Sydney Childrens Hosp Network, Sydney, NSW, Australia UZ Gent, Neuromuscular Reference Ctr, Ghent, BelgiumServais, L.论文数: 0 引用数: 0 h-index: 0机构: I Mot Hop Armand Trousseau, Paris, France UZ Gent, Neuromuscular Reference Ctr, Ghent, BelgiumVlodavets, D.论文数: 0 引用数: 0 h-index: 0机构: Pirogov Russian Natl Res Med Univ, Russian Children Neuromuscular Ctr, Veltischev Clin Pediat Res Inst, Moscow, Russia UZ Gent, Neuromuscular Reference Ctr, Ghent, BelgiumXiong, H.论文数: 0 引用数: 0 h-index: 0机构: Peking Univ First Hosp, Dept Pediat, Beijing, Peoples R China UZ Gent, Neuromuscular Reference Ctr, Ghent, BelgiumZanoteli, E.论文数: 0 引用数: 0 h-index: 0机构: Univ Sao Paulo FMUSP, Dept Neurol, Fac Med, Sao Paulo, Brazil UZ Gent, Neuromuscular Reference Ctr, Ghent, BelgiumEl-Khairi, M.论文数: 0 引用数: 0 h-index: 0机构: Roche Prod Ltd, Welwyn Garden City, Herts, England UZ Gent, Neuromuscular Reference Ctr, Ghent, BelgiumGerber, M.论文数: 0 引用数: 0 h-index: 0机构: Pharma Dev Safety F Hoffmann Roche Ltd, Basel, Switzerland UZ Gent, Neuromuscular Reference Ctr, Ghent, BelgiumGorni, K.论文数: 0 引用数: 0 h-index: 0机构: F Hoffmann Roche Ltd, PDMA Neurosci & Rare Dis, Basel, Switzerland UZ Gent, Neuromuscular Reference Ctr, Ghent, BelgiumKletzl, H.论文数: 0 引用数: 0 h-index: 0机构: Roche Innovat Ctr Basel, Roche Pharmaceut Res & Early Dev, Basel, Switzerland UZ Gent, Neuromuscular Reference Ctr, Ghent, BelgiumDodman, A.论文数: 0 引用数: 0 h-index: 0机构: F Hoffmann Roche Ltd, Pharma Dev Neurol, Basel, Switzerland UZ Gent, Neuromuscular Reference Ctr, Ghent, BelgiumGaki, E.论文数: 0 引用数: 0 h-index: 0机构: UZ Gent, Neuromuscular Reference Ctr, Ghent, BelgiumDarras, B.论文数: 0 引用数: 0 h-index: 0机构: UZ Gent, Neuromuscular Reference Ctr, Ghent, Belgium
- [34] Large deletions within the spinal muscular atrophy gene region in a patient with spinal muscular atrophy type 3NEURAL REGENERATION RESEARCH, 2011, 6 (23) : 1810 - 1813Wei, Wei论文数: 0 引用数: 0 h-index: 0机构: Zhejiang Univ Sch Med, Zhejiang Univ Adinovo Ctr Genet & Genom Med, Dept Biochem & Genet, Natl Educ Base Basic Med Sci,Inst Cell Biol, Hangzhou 310058, Zhejiang, Peoples R China Hangzhou Red Cross Hosp, Dept Orthoped, Hangzhou 310002, Zhejiang, Peoples R China Zhejiang Univ Sch Med, Zhejiang Univ Adinovo Ctr Genet & Genom Med, Dept Biochem & Genet, Natl Educ Base Basic Med Sci,Inst Cell Biol, Hangzhou 310058, Zhejiang, Peoples R ChinaChen, Chunyue论文数: 0 引用数: 0 h-index: 0机构: Zhejiang Univ Sch Med, Zhejiang Univ Adinovo Ctr Genet & Genom Med, Dept Biochem & Genet, Natl Educ Base Basic Med Sci,Inst Cell Biol, Hangzhou 310058, Zhejiang, Peoples R China Hangzhou Red Cross Hosp, Dept Orthoped, Hangzhou 310002, Zhejiang, Peoples R China Zhejiang Univ Sch Med, Zhejiang Univ Adinovo Ctr Genet & Genom Med, Dept Biochem & Genet, Natl Educ Base Basic Med Sci,Inst Cell Biol, Hangzhou 310058, Zhejiang, Peoples R ChinaLiu, Wenting论文数: 0 引用数: 0 h-index: 0机构: Zhejiang Univ Sch Med, Zhejiang Univ Adinovo Ctr Genet & Genom Med, Dept Biochem & Genet, Natl Educ Base Basic Med Sci,Inst Cell Biol, Hangzhou 310058, Zhejiang, Peoples R China Zhejiang Univ Sch Med, Zhejiang Univ Adinovo Ctr Genet & Genom Med, Dept Biochem & Genet, Natl Educ Base Basic Med Sci,Inst Cell Biol, Hangzhou 310058, Zhejiang, Peoples R ChinaDu, Zhenfang论文数: 0 引用数: 0 h-index: 0机构: Zhejiang Univ Sch Med, Zhejiang Univ Adinovo Ctr Genet & Genom Med, Dept Biochem & Genet, Natl Educ Base Basic Med Sci,Inst Cell Biol, Hangzhou 310058, Zhejiang, Peoples R China Zhejiang Univ Sch Med, Zhejiang Univ Adinovo Ctr Genet & Genom Med, Dept Biochem & Genet, Natl Educ Base Basic Med Sci,Inst Cell Biol, Hangzhou 310058, Zhejiang, Peoples R ChinaChen, Xiaoling论文数: 0 引用数: 0 h-index: 0机构: Zhejiang Univ Sch Med, Zhejiang Univ Adinovo Ctr Genet & Genom Med, Dept Biochem & Genet, Natl Educ Base Basic Med Sci,Inst Cell Biol, Hangzhou 310058, Zhejiang, Peoples R China Zhejiang Univ Sch Med, Zhejiang Univ Adinovo Ctr Genet & Genom Med, Dept Biochem & Genet, Natl Educ Base Basic Med Sci,Inst Cell Biol, Hangzhou 310058, Zhejiang, Peoples R ChinaZhang, Xianning论文数: 0 引用数: 0 h-index: 0机构: Zhejiang Univ Sch Med, Zhejiang Univ Adinovo Ctr Genet & Genom Med, Dept Biochem & Genet, Natl Educ Base Basic Med Sci,Inst Cell Biol, Hangzhou 310058, Zhejiang, Peoples R China Zhejiang Univ Sch Med, Zhejiang Univ Adinovo Ctr Genet & Genom Med, Dept Biochem & Genet, Natl Educ Base Basic Med Sci,Inst Cell Biol, Hangzhou 310058, Zhejiang, Peoples R China
- [35] Large deletions within the spinal muscular atrophy gene region in a patient with spinal muscular atrophy type 3Neural Regeneration Research, 2011, 6 (23) : 1810 - 1813Wei Wei1论文数: 0 引用数: 0 h-index: 0
- [36] Real-world evidence: Risdiplam in a patient with spinal muscular atrophy type I with a novel splicing mutation and one SMN2 copyHUMAN MOLECULAR GENETICS, 2024, 33 (13) : 1120 - 1130Ma, Kai论文数: 0 引用数: 0 h-index: 0机构: Shandong Univ, Pediat Res Inst, Childrens Hosp, Jingshi Rd 23976, Jinan 250022, Peoples R China Shandong Univ, Dept Neurol, Childrens Hosp, Jingshi Rd 23976, Jinan 250022, Peoples R China Shandong Univ, Pediat Res Inst, Childrens Hosp, Jingshi Rd 23976, Jinan 250022, Peoples R ChinaZhang, Kaihui论文数: 0 引用数: 0 h-index: 0机构: Shandong Univ, Pediat Res Inst, Childrens Hosp, Jingshi Rd 23976, Jinan 250022, Peoples R China Shandong Univ, Pediat Res Inst, Childrens Hosp, Jingshi Rd 23976, Jinan 250022, Peoples R ChinaChen, Defang论文数: 0 引用数: 0 h-index: 0机构: Shandong First Med Univ, Off Operat Management Comm, Cent Hosp, Jiefang Rd 105, Jinan 250022, Peoples R China Shandong Univ, Pediat Res Inst, Childrens Hosp, Jingshi Rd 23976, Jinan 250022, Peoples R ChinaWang, Chuan论文数: 0 引用数: 0 h-index: 0机构: Shandong Univ, Childrens Hosp, Sci Educ & Foreign Affairs Sect, Jingshi Rd 23976, Jinan 250022, Peoples R China Shandong Univ, Pediat Res Inst, Childrens Hosp, Jingshi Rd 23976, Jinan 250022, Peoples R ChinaAbdalla, Mohnad论文数: 0 引用数: 0 h-index: 0机构: Shandong Univ, Pediat Res Inst, Childrens Hosp, Jingshi Rd 23976, Jinan 250022, Peoples R China Shandong Univ, Pediat Res Inst, Childrens Hosp, Jingshi Rd 23976, Jinan 250022, Peoples R ChinaZhang, Haozheng论文数: 0 引用数: 0 h-index: 0机构: Shandong Univ, Pediat Res Inst, Childrens Hosp, Jingshi Rd 23976, Jinan 250022, Peoples R China Shandong Univ, Pediat Res Inst, Childrens Hosp, Jingshi Rd 23976, Jinan 250022, Peoples R ChinaTian, Rujin论文数: 0 引用数: 0 h-index: 0机构: Shandong Univ, Pediat Res Inst, Childrens Hosp, Jingshi Rd 23976, Jinan 250022, Peoples R China Shandong Univ, Pediat Res Inst, Childrens Hosp, Jingshi Rd 23976, Jinan 250022, Peoples R ChinaLiu, Yang论文数: 0 引用数: 0 h-index: 0机构: Shandong Univ, Pediat Res Inst, Childrens Hosp, Jingshi Rd 23976, Jinan 250022, Peoples R China Shandong Univ, Ophthalmol Dept, Childrens Hosp, Jingshi Rd 23976, Jinan 250022, Peoples R China Shandong Univ, Pediat Res Inst, Childrens Hosp, Jingshi Rd 23976, Jinan 250022, Peoples R China论文数: 引用数: h-index:机构:Zhang, Xinyi论文数: 0 引用数: 0 h-index: 0机构: Second Peoples Hosp Shandong Prov, Intens Care Unit, Duanxing West Rd 4, Jinan 250022, Peoples R China Shandong Univ, Pediat Res Inst, Childrens Hosp, Jingshi Rd 23976, Jinan 250022, Peoples R ChinaLiu, Fangfang论文数: 0 引用数: 0 h-index: 0机构: Shandong First Med Univ, Dept Ultrasound, Cent Hosp, Jiefang Rd 105, Jinan 250022, Peoples R China Shandong Univ, Pediat Res Inst, Childrens Hosp, Jingshi Rd 23976, Jinan 250022, Peoples R ChinaLiu, Guohua论文数: 0 引用数: 0 h-index: 0机构: Shandong Univ, Ophthalmol Dept, Childrens Hosp, Jingshi Rd 23976, Jinan 250022, Peoples R China Shandong Univ, Pediat Res Inst, Childrens Hosp, Jingshi Rd 23976, Jinan 250022, Peoples R ChinaWang, Dong论文数: 0 引用数: 0 h-index: 0机构: Shandong Univ, Pediat Res Inst, Childrens Hosp, Jingshi Rd 23976, Jinan 250022, Peoples R China Shandong Univ, Pediat Res Inst, Childrens Hosp, Jingshi Rd 23976, Jinan 250022, Peoples R China
- [37] Is treatment with oral risdiplam effective and well-tolerated for infants with spinal muscular atrophy type 1?JOURNAL OF PERINATOLOGY, 2022, 42 (05) : 689 - 691Gaffar, Sheema论文数: 0 引用数: 0 h-index: 0机构: Univ Southern Calif, Div Neonatol, Dept Pediat, LAC USC Med Ctr,Keck Sch Med, Los Angeles, CA 90007 USA Univ Southern Calif, Div Neonatol, Dept Pediat, LAC USC Med Ctr,Keck Sch Med, Los Angeles, CA 90007 USA论文数: 引用数: h-index:机构:Ramanathan, Rangasamy论文数: 0 引用数: 0 h-index: 0机构: Univ Southern Calif, Div Neonatol, Dept Pediat, LAC USC Med Ctr,Keck Sch Med, Los Angeles, CA 90007 USA Univ Southern Calif, Div Neonatol, Dept Pediat, LAC USC Med Ctr,Keck Sch Med, Los Angeles, CA 90007 USA
- [38] Is treatment with oral risdiplam effective and well-tolerated for infants with spinal muscular atrophy type 1?Journal of Perinatology, 2022, 42 : 689 - 691Sheema Gaffar论文数: 0 引用数: 0 h-index: 0机构: University of Southern California,Division of Neonatology, Department of Pediatrics, LAC+USC Medical Center, Keck School of MedicineRowena Cayabyab论文数: 0 引用数: 0 h-index: 0机构: University of Southern California,Division of Neonatology, Department of Pediatrics, LAC+USC Medical Center, Keck School of MedicineRangasamy Ramanathan论文数: 0 引用数: 0 h-index: 0机构: University of Southern California,Division of Neonatology, Department of Pediatrics, LAC+USC Medical Center, Keck School of Medicine
- [39] Risdiplam in Patients Previously Treated with Other Therapies for Spinal Muscular Atrophy: An Interim Analysis from the JEWELFISH StudyNeurology and Therapy, 2023, 12 : 543 - 557Claudia A. Chiriboga论文数: 0 引用数: 0 h-index: 0机构: Columbia University Irving Medical Center,Department of NeurologyClaudio Bruno论文数: 0 引用数: 0 h-index: 0机构: Columbia University Irving Medical Center,Department of NeurologyTina Duong论文数: 0 引用数: 0 h-index: 0机构: Columbia University Irving Medical Center,Department of NeurologyDirk Fischer论文数: 0 引用数: 0 h-index: 0机构: Columbia University Irving Medical Center,Department of NeurologyEugenio Mercuri论文数: 0 引用数: 0 h-index: 0机构: Columbia University Irving Medical Center,Department of NeurologyJanbernd Kirschner论文数: 0 引用数: 0 h-index: 0机构: Columbia University Irving Medical Center,Department of NeurologyAnna Kostera-Pruszczyk论文数: 0 引用数: 0 h-index: 0机构: Columbia University Irving Medical Center,Department of NeurologyBirgit Jaber论文数: 0 引用数: 0 h-index: 0机构: Columbia University Irving Medical Center,Department of NeurologyKsenija Gorni论文数: 0 引用数: 0 h-index: 0机构: Columbia University Irving Medical Center,Department of NeurologyHeidemarie Kletzl论文数: 0 引用数: 0 h-index: 0机构: Columbia University Irving Medical Center,Department of NeurologyImogen Carruthers论文数: 0 引用数: 0 h-index: 0机构: Columbia University Irving Medical Center,Department of NeurologyCarmen Martin论文数: 0 引用数: 0 h-index: 0机构: Columbia University Irving Medical Center,Department of NeurologyFrancis Warren论文数: 0 引用数: 0 h-index: 0机构: Columbia University Irving Medical Center,Department of NeurologyRenata S. Scalco论文数: 0 引用数: 0 h-index: 0机构: Columbia University Irving Medical Center,Department of NeurologyKathryn R. Wagner论文数: 0 引用数: 0 h-index: 0机构: Columbia University Irving Medical Center,Department of NeurologyFrancesco Muntoni论文数: 0 引用数: 0 h-index: 0机构: Columbia University Irving Medical Center,Department of Neurology
- [40] RAINBOWFISH: Preliminary efficacy and safety data in risdiplam-treated infants with presymptomatic spinal muscular atrophy (SMA)NEUROMUSCULAR DISORDERS, 2022, 32 : S85 - S86Finkel, R.论文数: 0 引用数: 0 h-index: 0机构: St Judes Res Hosp, Memphis, TN USA St Judes Res Hosp, Memphis, TN USAFarrar, M.论文数: 0 引用数: 0 h-index: 0机构: Sydney Childrens Hosp, Sydney, NSW, Australia St Judes Res Hosp, Memphis, TN USAVlodavets, D.论文数: 0 引用数: 0 h-index: 0机构: Pirogov Med Univ, Moscow, Russia St Judes Res Hosp, Memphis, TN USAZanoteli, E.论文数: 0 引用数: 0 h-index: 0机构: Univ Sao Paulo, Sao Paulo, Brazil St Judes Res Hosp, Memphis, TN USAAl-Muhaizea, M.论文数: 0 引用数: 0 h-index: 0机构: King Faisal Specialist Hosp & Res Ctr, Riyadh, Saudi Arabia St Judes Res Hosp, Memphis, TN USANelson, L.论文数: 0 引用数: 0 h-index: 0机构: UT SouthWestern Med Ctr, Dallas, TX USA St Judes Res Hosp, Memphis, TN USAPrufer, A.论文数: 0 引用数: 0 h-index: 0机构: Fed Uni Rio De Janeiro, Rio De Janeiro, Brazil St Judes Res Hosp, Memphis, TN USA论文数: 引用数: h-index:机构:Wang, Y.论文数: 0 引用数: 0 h-index: 0机构: Childrens Hosp Fudan, Shanghai, Peoples R China St Judes Res Hosp, Memphis, TN USAFisher, C.论文数: 0 引用数: 0 h-index: 0机构: Roche Prod Ltd, Welwyn Garden City, Herts, England St Judes Res Hosp, Memphis, TN USAGerber, M.论文数: 0 引用数: 0 h-index: 0机构: St Judes Res Hosp, Memphis, TN USAGorni, K.论文数: 0 引用数: 0 h-index: 0机构: F Hoffmann La Roche Ltd, Basel, Switzerland St Judes Res Hosp, Memphis, TN USAKletzl, H.论文数: 0 引用数: 0 h-index: 0机构: Roche Innovat Ctr Basel, Basel, Switzerland St Judes Res Hosp, Memphis, TN USAPalfreeman, L.论文数: 0 引用数: 0 h-index: 0机构: Roche Prod Ltd, Welwyn Garden City, Herts, England St Judes Res Hosp, Memphis, TN USAScalco, R.论文数: 0 引用数: 0 h-index: 0机构: F Hoffmann La Roche Ltd, Basel, Switzerland St Judes Res Hosp, Memphis, TN USABertini, E.论文数: 0 引用数: 0 h-index: 0机构: Res Hosp IRCCS, Rome, Italy St Judes Res Hosp, Memphis, TN USA