共 57 条
- [1] Albrechtsen SS, 2020, DAN MED J, V67
- [2] Spinal Muscular Atrophy Treatment in Patients Identified by Newborn Screening-A Systematic Review[J]. GENES, 2023, 14 (07)论文数: 引用数: h-index:机构:Mouraux, Charlotte论文数: 0 引用数: 0 h-index: 0机构: Univ Liege, Univ Hosp Liege, Neuromuscular Reference Ctr, Dept Pediat, B-4000 Liege, Belgium Med Univ Warsaw, Dept Neurol, PL-02097 Warsaw, PolandDangouloff, Tamara论文数: 0 引用数: 0 h-index: 0机构: Univ Liege, Univ Hosp Liege, Neuromuscular Reference Ctr, Dept Pediat, B-4000 Liege, Belgium Med Univ Warsaw, Dept Neurol, PL-02097 Warsaw, PolandServais, Laurent论文数: 0 引用数: 0 h-index: 0机构: Univ Liege, Univ Hosp Liege, Neuromuscular Reference Ctr, Dept Pediat, B-4000 Liege, Belgium Univ Oxford, MDUK Oxford Neuromuscular Ctr & NIHR Oxford Biomed, Oxford OX3 0ER, England Med Univ Warsaw, Dept Neurol, PL-02097 Warsaw, Poland
- [3] Onasemnogene Abeparvovec: A Review in Spinal Muscular Atrophy[J]. CNS DRUGS, 2022, 36 (09) : 995 - 1005Blair, Hannah A.论文数: 0 引用数: 0 h-index: 0机构: Springer Nat, Private Bag 65901, Auckland 0754, New Zealand Springer Nat, Private Bag 65901, Auckland 0754, New Zealand
- [4] Newborn Screening for SMA - Can a Wait-and-See Strategy be Responsibly Justified in Patients With Four SMN2 Copies?[J]. JOURNAL OF NEUROMUSCULAR DISEASES, 2022, 9 (05) : 597 - 605Blaschek, Astrid论文数: 0 引用数: 0 h-index: 0机构: Ludwig Maximilians Univ Munchen, Dr von Hauner Childrens Hosp, LMU Hosp, Dept Pediat Neurol & Dev Med M, Munich, Germany Ludwig Maximilians Univ Munchen, Dr von Hauner Childrens Hosp, LMU Hosp, LMU Ctr Children Med Complex, Munich, Germany Ludwig Maximilians Univ Munchen, Dr von Hauner Childrens Hosp, LMU Hosp, Dept Pediat Neurol & Dev Med M, Munich, GermanyKoelbel, Heike论文数: 0 引用数: 0 h-index: 0机构: Univ Essen Gesamthsch, Dept Pediat Neurol, Dev Neurol & Social Pediat, Essen, Germany Ludwig Maximilians Univ Munchen, Dr von Hauner Childrens Hosp, LMU Hosp, Dept Pediat Neurol & Dev Med M, Munich, GermanySchwartz, Oliver论文数: 0 引用数: 0 h-index: 0机构: Munster Univ Hosp, Dept Pediat Neurol, Munster, Germany Ludwig Maximilians Univ Munchen, Dr von Hauner Childrens Hosp, LMU Hosp, Dept Pediat Neurol & Dev Med M, Munich, GermanyKoehler, Cornelia论文数: 0 引用数: 0 h-index: 0机构: Univ Bochum, Dept Pediat Neurol, Bochum, Germany Ludwig Maximilians Univ Munchen, Dr von Hauner Childrens Hosp, LMU Hosp, Dept Pediat Neurol & Dev Med M, Munich, GermanyGlaeser, Dieter论文数: 0 引用数: 0 h-index: 0机构: MVZ Genetikum GmbH, Ctr Human Genet, Neu Ulm, Germany Ludwig Maximilians Univ Munchen, Dr von Hauner Childrens Hosp, LMU Hosp, Dept Pediat Neurol & Dev Med M, Munich, GermanyEggermann, Katja论文数: 0 引用数: 0 h-index: 0机构: Rhein Westfal TH Aachen, Med Fac, Inst Human Genet, Aachen, Germany Ludwig Maximilians Univ Munchen, Dr von Hauner Childrens Hosp, LMU Hosp, Dept Pediat Neurol & Dev Med M, Munich, GermanyHannibal, Iris论文数: 0 引用数: 0 h-index: 0机构: Ludwig Maximilians Univ Munchen, Dr von Hauner Childrens Hosp, LMU Hosp, Dept Pediat Neurol & Dev Med M, Munich, Germany Ludwig Maximilians Univ Munchen, Dr von Hauner Childrens Hosp, LMU Hosp, LMU Ctr Children Med Complex, Munich, Germany Ludwig Maximilians Univ Munchen, Dr von Hauner Childrens Hosp, LMU Hosp, Dept Pediat Neurol & Dev Med M, Munich, GermanySchara-Schmidt, Ulrike论文数: 0 引用数: 0 h-index: 0机构: Univ Essen Gesamthsch, Dept Pediat Neurol, Dev Neurol & Social Pediat, Essen, Germany Ludwig Maximilians Univ Munchen, Dr von Hauner Childrens Hosp, LMU Hosp, Dept Pediat Neurol & Dev Med M, Munich, GermanyMueller-Felber, Wolfgang论文数: 0 引用数: 0 h-index: 0机构: Ludwig Maximilians Univ Munchen, Dr von Hauner Childrens Hosp, LMU Hosp, Dept Pediat Neurol & Dev Med M, Munich, Germany Ludwig Maximilians Univ Munchen, Dr von Hauner Childrens Hosp, LMU Hosp, LMU Ctr Children Med Complex, Munich, Germany Ludwig Maximilians Univ Munchen, Dr von Hauner Childrens Hosp, LMU Hosp, Dept Pediat Neurol & Dev Med M, Munich, GermanyVill, Katharina论文数: 0 引用数: 0 h-index: 0机构: Ludwig Maximilians Univ Munchen, Dr von Hauner Childrens Hosp, LMU Hosp, Dept Pediat Neurol & Dev Med M, Munich, Germany Ludwig Maximilians Univ Munchen, Dr von Hauner Childrens Hosp, LMU Hosp, LMU Ctr Children Med Complex, Munich, Germany Ludwig Maximilians Univ Munchen, Dr von Hauner Childrens Hosp, LMU Hosp, Dept Pediat Neurol & Dev Med M, Munich, Germany
- [5] Three years pilot of spinal muscular atrophy newborn screening turned into official program in Southern Belgium[J]. SCIENTIFIC REPORTS, 2021, 11 (01)Boemer, Francois论文数: 0 引用数: 0 h-index: 0机构: Univ Liege, CHU Sart Tilman, Biochem Genet Lab, Human Genet,B35, B-4000 Liege, Belgium Univ Liege, CHU Sart Tilman, Biochem Genet Lab, Human Genet,B35, B-4000 Liege, BelgiumCaberg, Jean-Hubert论文数: 0 引用数: 0 h-index: 0机构: Univ Liege, CHU Liege, Dept Human Genet, Mol Genet Lab, Liege, Belgium Univ Liege, CHU Sart Tilman, Biochem Genet Lab, Human Genet,B35, B-4000 Liege, BelgiumBeckers, Pablo论文数: 0 引用数: 0 h-index: 0机构: Univ Liege, CHU Sart Tilman, Biochem Genet Lab, Human Genet,B35, B-4000 Liege, Belgium Univ Liege, CHU Sart Tilman, Biochem Genet Lab, Human Genet,B35, B-4000 Liege, BelgiumDideberg, Vinciane论文数: 0 引用数: 0 h-index: 0机构: Univ Liege, CHU Liege, Dept Human Genet, Mol Genet Lab, Liege, Belgium Univ Liege, CHU Sart Tilman, Biochem Genet Lab, Human Genet,B35, B-4000 Liege, Belgiumdi Fiore, Samantha论文数: 0 引用数: 0 h-index: 0机构: Univ Liege, CHU Sart Tilman, Biochem Genet Lab, Human Genet,B35, B-4000 Liege, Belgium Univ Liege, CHU Sart Tilman, Biochem Genet Lab, Human Genet,B35, B-4000 Liege, BelgiumBours, Vincent论文数: 0 引用数: 0 h-index: 0机构: Univ Liege, CHU Liege, Dept Human Genet, Liege, Belgium Univ Liege, CHU Sart Tilman, Biochem Genet Lab, Human Genet,B35, B-4000 Liege, BelgiumMarie, Sandrine论文数: 0 引用数: 0 h-index: 0机构: Clin Univ St Luc, UCLouvain, Dept Lab Med, Brussels, Belgium Univ Liege, CHU Sart Tilman, Biochem Genet Lab, Human Genet,B35, B-4000 Liege, BelgiumDewulf, Joseph论文数: 0 引用数: 0 h-index: 0机构: Clin Univ St Luc, UCLouvain, Dept Lab Med, Brussels, Belgium Univ Liege, CHU Sart Tilman, Biochem Genet Lab, Human Genet,B35, B-4000 Liege, BelgiumMarcelis, Lionel论文数: 0 引用数: 0 h-index: 0机构: Free Univ Brussels, Lab Pediat Res, Brussels, Belgium Univ Liege, CHU Sart Tilman, Biochem Genet Lab, Human Genet,B35, B-4000 Liege, BelgiumDeconinck, Nicolas论文数: 0 引用数: 0 h-index: 0机构: Univ Libre Bruxelles, Hop Enfants Reine Fabiola HUDERF, Neuromuscular Reference Ctr, Brussels, Belgium Univ Liege, CHU Sart Tilman, Biochem Genet Lab, Human Genet,B35, B-4000 Liege, BelgiumDaron, Aurore论文数: 0 引用数: 0 h-index: 0机构: Univ Liege, CHR Citadelle, Dept Pediat, Reference Ctr Neuromuscular Dis,Div Child Neurol, Liege, Belgium Univ Liege, CHU Sart Tilman, Biochem Genet Lab, Human Genet,B35, B-4000 Liege, BelgiumBlasco-Perez, Laura论文数: 0 引用数: 0 h-index: 0机构: Hosp Valle De Hebron, Dept Clin & Mol Genet, Barcelona, Spain Univ Liege, CHU Sart Tilman, Biochem Genet Lab, Human Genet,B35, B-4000 Liege, BelgiumTizzano, Eduardo论文数: 0 引用数: 0 h-index: 0机构: Hosp Valle De Hebron, Dept Clin & Mol Genet, Barcelona, Spain Univ Liege, CHU Sart Tilman, Biochem Genet Lab, Human Genet,B35, B-4000 Liege, Belgium论文数: 引用数: h-index:机构:Lombet, Jacques论文数: 0 引用数: 0 h-index: 0机构: ONE, Off Naissance & Enfance, Herstal, Belgium Univ Liege, CHU Sart Tilman, Biochem Genet Lab, Human Genet,B35, B-4000 Liege, BelgiumPereira, Tatiana论文数: 0 引用数: 0 h-index: 0机构: ONE, Off Naissance & Enfance, Herstal, Belgium Univ Liege, CHU Sart Tilman, Biochem Genet Lab, Human Genet,B35, B-4000 Liege, BelgiumLopez-Granados, Lucia论文数: 0 引用数: 0 h-index: 0机构: ONE, Off Naissance & Enfance, Herstal, Belgium Univ Liege, CHU Sart Tilman, Biochem Genet Lab, Human Genet,B35, B-4000 Liege, BelgiumShalchian-Tehran, Sarvnaz论文数: 0 引用数: 0 h-index: 0机构: CHR Citadelle, Dept Neurol & Eth Comm, Liege, Belgium Univ Liege, CHU Sart Tilman, Biochem Genet Lab, Human Genet,B35, B-4000 Liege, Belgiumvan Assche, Veronique论文数: 0 引用数: 0 h-index: 0机构: Assoc Belge Malad Neuromsculaires ASBL, ABMM, La Louviere, Belgium Univ Liege, CHU Sart Tilman, Biochem Genet Lab, Human Genet,B35, B-4000 Liege, BelgiumWillems, Arabelle论文数: 0 引用数: 0 h-index: 0机构: Assoc Belge Malad Neuromsculaires ASBL, ABMM, La Louviere, Belgium Univ Liege, CHU Sart Tilman, Biochem Genet Lab, Human Genet,B35, B-4000 Liege, BelgiumHuybrechts, Sofie论文数: 0 引用数: 0 h-index: 0机构: Biogen, Cambridge, MA USA Univ Liege, CHU Sart Tilman, Biochem Genet Lab, Human Genet,B35, B-4000 Liege, BelgiumMast, Benedicte论文数: 0 引用数: 0 h-index: 0机构: Roche, Basel, Switzerland Univ Liege, CHU Sart Tilman, Biochem Genet Lab, Human Genet,B35, B-4000 Liege, Belgiumvan Olden, Rudolf论文数: 0 引用数: 0 h-index: 0机构: Novartis Gene Therapies Switzerland GmBH, Zurich, Switzerland Univ Liege, CHU Sart Tilman, Biochem Genet Lab, Human Genet,B35, B-4000 Liege, BelgiumDangouloff, Tamara论文数: 0 引用数: 0 h-index: 0机构: Univ Liege, CHR Citadelle, Dept Pediat, Reference Ctr Neuromuscular Dis,Div Child Neurol, Liege, Belgium Univ Liege, CHU Sart Tilman, Biochem Genet Lab, Human Genet,B35, B-4000 Liege, BelgiumServais, Laurent论文数: 0 引用数: 0 h-index: 0机构: Univ Liege, CHR Citadelle, Dept Pediat, Reference Ctr Neuromuscular Dis,Div Child Neurol, Liege, Belgium Univ Oxford, Dept Paediat, MDUK Neuromuscular Ctr, Oxford, England Univ Liege, CHU Sart Tilman, Biochem Genet Lab, Human Genet,B35, B-4000 Liege, Belgium
- [6] Onasemnogene Abeparvovec for Treating Pre-symptomatic Spinal Muscular Atrophy: An External Assessment Group Perspective of the Partial Review of NICE Highly Specialised Technology Evaluation 15[J]. PHARMACOECONOMICS-OPEN, 2023, 7 (06) : 863 - 875Chaplin, Marty论文数: 0 引用数: 0 h-index: 0机构: Univ Liverpool, Liverpool Reviews & Implementat Grp, Whelan Bldg,Brownlow Hill, Liverpool L69 3GB, England Univ Liverpool, Liverpool Reviews & Implementat Grp, Whelan Bldg,Brownlow Hill, Liverpool L69 3GB, EnglandBresnahan, Rebecca论文数: 0 引用数: 0 h-index: 0机构: Univ Liverpool, Liverpool Reviews & Implementat Grp, Whelan Bldg,Brownlow Hill, Liverpool L69 3GB, England Univ Liverpool, Liverpool Reviews & Implementat Grp, Whelan Bldg,Brownlow Hill, Liverpool L69 3GB, England论文数: 引用数: h-index:机构:Mahon, James论文数: 0 引用数: 0 h-index: 0机构: Coldingham Analyt Serv, Eyemouth, Berwick, England Univ Liverpool, Liverpool Reviews & Implementat Grp, Whelan Bldg,Brownlow Hill, Liverpool L69 3GB, EnglandHouten, Rachel论文数: 0 引用数: 0 h-index: 0机构: Univ Liverpool, Liverpool Reviews & Implementat Grp, Whelan Bldg,Brownlow Hill, Liverpool L69 3GB, England Univ Liverpool, Liverpool Reviews & Implementat Grp, Whelan Bldg,Brownlow Hill, Liverpool L69 3GB, EnglandBeale, Sophie论文数: 0 引用数: 0 h-index: 0机构: Hare Res, York, N Yorkshire, England Univ Liverpool, Liverpool Reviews & Implementat Grp, Whelan Bldg,Brownlow Hill, Liverpool L69 3GB, England论文数: 引用数: h-index:机构:Dundar, Yenal论文数: 0 引用数: 0 h-index: 0机构: Univ Liverpool, Liverpool Reviews & Implementat Grp, Whelan Bldg,Brownlow Hill, Liverpool L69 3GB, England Univ Liverpool, Liverpool Reviews & Implementat Grp, Whelan Bldg,Brownlow Hill, Liverpool L69 3GB, EnglandMarsden, Ashley论文数: 0 引用数: 0 h-index: 0机构: North West Med Informat Ctr, Liverpool, England Univ Liverpool, Liverpool Reviews & Implementat Grp, Whelan Bldg,Brownlow Hill, Liverpool L69 3GB, EnglandMunot, Pinki论文数: 0 引用数: 0 h-index: 0机构: Great Ormond Hosp Children NHS Fdn Trust, London, England Univ Liverpool, Liverpool Reviews & Implementat Grp, Whelan Bldg,Brownlow Hill, Liverpool L69 3GB, England
- [7] Pharmacotherapy for Spinal Muscular Atrophy in Babies and Children: A Review of Approved and Experimental Therapies[J]. PEDIATRIC DRUGS, 2022, 24 (06) : 585 - 602Chiriboga, Claudia A.论文数: 0 引用数: 0 h-index: 0机构: Columbia Univ, Dept Neurol, Div Child Neurol, Med Ctr, 180 Ft Washington Ave, New York, NY 10032 USA Columbia Univ, Dept Neurol, Div Child Neurol, Med Ctr, 180 Ft Washington Ave, New York, NY 10032 USA
- [8] ClinicalTrials.gov, A Study of Risdiplam in Infants with Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy (RAINBOWFISH)
- [9] Continued benefit of nusinersen initiated in the presymptomatic stage of spinal muscular atrophy: 5-year update of the NURTURE study[J]. MUSCLE & NERVE, 2023, 68 (02) : 157 - 170Crawford, Thomas J.论文数: 0 引用数: 0 h-index: 0机构: Johns Hopkins Univ, Sch Med, Dept Neurol, 200 North Wolfe St,Rubenstein Child Hlth Bldg 214, Baltimore, MD 21287 USA Johns Hopkins Univ, Sch Med, Dept Neurol, Baltimore, MD USA Johns Hopkins Univ, Sch Med, Dept Neurol, 200 North Wolfe St,Rubenstein Child Hlth Bldg 214, Baltimore, MD 21287 USASwoboda, Kathryn C.论文数: 0 引用数: 0 h-index: 0机构: Massachusetts Gen Hosp, Ctr Genom Med, Dept Neurol, Boston, MA USA Johns Hopkins Univ, Sch Med, Dept Neurol, 200 North Wolfe St,Rubenstein Child Hlth Bldg 214, Baltimore, MD 21287 USADe Vivo, Darryl论文数: 0 引用数: 0 h-index: 0机构: Columbia Univ, Irving Med Ctr, Dept Neurol, New York, NY USA Columbia Univ, Irving Med Ctr, Dept Pediat, New York, NY USA Johns Hopkins Univ, Sch Med, Dept Neurol, 200 North Wolfe St,Rubenstein Child Hlth Bldg 214, Baltimore, MD 21287 USABertini, Enrico论文数: 0 引用数: 0 h-index: 0机构: Postgrad Bambino Gesu Childrens Res Hosp, IRCCS, Unit Neuromuscular & Neurodegenerat Disorders, Rome, Italy Johns Hopkins Univ, Sch Med, Dept Neurol, 200 North Wolfe St,Rubenstein Child Hlth Bldg 214, Baltimore, MD 21287 USAHwu, Wuh-Liang S.论文数: 0 引用数: 0 h-index: 0机构: Natl Taiwan Univ Hosp, Dept Med Genet, Taipei, Taiwan Natl Taiwan Univ Hosp, Dept Pediat, Taipei, Taiwan Johns Hopkins Univ, Sch Med, Dept Neurol, 200 North Wolfe St,Rubenstein Child Hlth Bldg 214, Baltimore, MD 21287 USAFinkel, Richard论文数: 0 引用数: 0 h-index: 0机构: St Jude Childrens Res Hosp, Ctr Expt Neurotherapeut, Memphis, TN USA Nemours Childrens Hosp, Div Neurol, Orlando, FL USA Johns Hopkins Univ, Sch Med, Dept Neurol, 200 North Wolfe St,Rubenstein Child Hlth Bldg 214, Baltimore, MD 21287 USAKirschner, Janbernd L.论文数: 0 引用数: 0 h-index: 0机构: Univ Freiburg, Fac Med, Dept Neuropediat & Muscle Disorders, Freiburg, Germany Johns Hopkins Univ, Sch Med, Dept Neurol, 200 North Wolfe St,Rubenstein Child Hlth Bldg 214, Baltimore, MD 21287 USAKuntz, Nancy论文数: 0 引用数: 0 h-index: 0机构: Ann & Robert H Lurie Childrens Hosp Chicago, Chicago, IL USA Johns Hopkins Univ, Sch Med, Dept Neurol, 200 North Wolfe St,Rubenstein Child Hlth Bldg 214, Baltimore, MD 21287 USANazario, Aledie Navas A.论文数: 0 引用数: 0 h-index: 0机构: Nemours Childrens Hosp, Dept Pediat, Div Pulm Med, Orlando, FL USA Johns Hopkins Univ, Sch Med, Dept Neurol, 200 North Wolfe St,Rubenstein Child Hlth Bldg 214, Baltimore, MD 21287 USAParsons, Julie论文数: 0 引用数: 0 h-index: 0机构: Univ Colorado, Childrens Hosp Colorado, Sch Med, Aurora, CO USA Johns Hopkins Univ, Sch Med, Dept Neurol, 200 North Wolfe St,Rubenstein Child Hlth Bldg 214, Baltimore, MD 21287 USAPechmann, Astrid M.论文数: 0 引用数: 0 h-index: 0机构: Univ Freiburg, Fac Med, Dept Neuropediat & Muscle Disorders, Freiburg, Germany Johns Hopkins Univ, Sch Med, Dept Neurol, 200 North Wolfe St,Rubenstein Child Hlth Bldg 214, Baltimore, MD 21287 USARyan, M. Monique M. J.论文数: 0 引用数: 0 h-index: 0机构: Murdoch Childrens Res Inst, Melbourne, Australia Univ Melbourne, Melbourne, Australia Johns Hopkins Univ, Sch Med, Dept Neurol, 200 North Wolfe St,Rubenstein Child Hlth Bldg 214, Baltimore, MD 21287 USA论文数: 引用数: h-index:机构:Topaloglu, Haluk论文数: 0 引用数: 0 h-index: 0机构: Hacettepe Univ, Dept Pediat Neurol, Ankara, Turkiye Johns Hopkins Univ, Sch Med, Dept Neurol, 200 North Wolfe St,Rubenstein Child Hlth Bldg 214, Baltimore, MD 21287 USABen-Omran, Tawfeg A.论文数: 0 引用数: 0 h-index: 0机构: Sidra Med, Div Genet & Genom Med, Doha, Qatar Hamad Med Corp, Med Genet Dept, Doha, Qatar Johns Hopkins Univ, Sch Med, Dept Neurol, 200 North Wolfe St,Rubenstein Child Hlth Bldg 214, Baltimore, MD 21287 USASansone, Valeria论文数: 0 引用数: 0 h-index: 0机构: Univ Milan, NeMo Clin Ctr, Neurorehabil Unit, Milan, Italy Univ Milan, Dept Biomed Sci Hlth, Milan, Italy Johns Hopkins Univ, Sch Med, Dept Neurol, 200 North Wolfe St,Rubenstein Child Hlth Bldg 214, Baltimore, MD 21287 USAJong, Yuh-Jyh论文数: 0 引用数: 0 h-index: 0机构: Kaohsiung Med Univ, Kaohsiung Med Univ Hosp, Grad Inst Clin Med, Coll Med, Kaohsiung, Taiwan Kaohsiung Med Univ, Kaohsiung Med Univ Hosp, Translat Res Ctr Neuromuscular Dis, Kaohsiung, Taiwan Kaohsiung Med Univ, Kaohsiung Med Univ Hosp, Dept Pediat, Kaohsiung, Taiwan Kaohsiung Med Univ, Kaohsiung Med Univ Hosp, Dept Lab Med, Kaohsiung, Taiwan Johns Hopkins Univ, Sch Med, Dept Neurol, 200 North Wolfe St,Rubenstein Child Hlth Bldg 214, Baltimore, MD 21287 USAShu, Francy论文数: 0 引用数: 0 h-index: 0机构: UCLA, David Geffen Sch Med, Dept Neurol, Los Angeles, CA USA Johns Hopkins Univ, Sch Med, Dept Neurol, 200 North Wolfe St,Rubenstein Child Hlth Bldg 214, Baltimore, MD 21287 USAZhu, Cong论文数: 0 引用数: 0 h-index: 0机构: Biogen, Cambridge, MA USA Johns Hopkins Univ, Sch Med, Dept Neurol, 200 North Wolfe St,Rubenstein Child Hlth Bldg 214, Baltimore, MD 21287 USARaynaud, Stephanie R.论文数: 0 引用数: 0 h-index: 0机构: Biogen, Cambridge, MA USA Johns Hopkins Univ, Sch Med, Dept Neurol, 200 North Wolfe St,Rubenstein Child Hlth Bldg 214, Baltimore, MD 21287 USALago, Tiffany D.论文数: 0 引用数: 0 h-index: 0机构: Biogen, Cambridge, MA USA Johns Hopkins Univ, Sch Med, Dept Neurol, 200 North Wolfe St,Rubenstein Child Hlth Bldg 214, Baltimore, MD 21287 USAParadis, Angela论文数: 0 引用数: 0 h-index: 0机构: Biogen, Cambridge, MA USA Johns Hopkins Univ, Sch Med, Dept Neurol, 200 North Wolfe St,Rubenstein Child Hlth Bldg 214, Baltimore, MD 21287 USAFoster, Richard论文数: 0 引用数: 0 h-index: 0机构: Biogen, Maidenhead, Berks, England Johns Hopkins Univ, Sch Med, Dept Neurol, 200 North Wolfe St,Rubenstein Child Hlth Bldg 214, Baltimore, MD 21287 USAChin, Russell论文数: 0 引用数: 0 h-index: 0机构: Biogen, Cambridge, MA USA Johns Hopkins Univ, Sch Med, Dept Neurol, 200 North Wolfe St,Rubenstein Child Hlth Bldg 214, Baltimore, MD 21287 USABerger, Zdenek论文数: 0 引用数: 0 h-index: 0机构: Biogen, 300 Binney St, Cambridge, MA 02142 USA Biogen, Cambridge, MA USA Johns Hopkins Univ, Sch Med, Dept Neurol, 200 North Wolfe St,Rubenstein Child Hlth Bldg 214, Baltimore, MD 21287 USANURTURE Study Grp论文数: 0 引用数: 0 h-index: 0机构: Johns Hopkins Univ, Sch Med, Dept Neurol, 200 North Wolfe St,Rubenstein Child Hlth Bldg 214, Baltimore, MD 21287 USA
- [10] Financial cost and quality of life of patients with spinal muscular atrophy identified by symptoms or newborn screening[J]. DEVELOPMENTAL MEDICINE AND CHILD NEUROLOGY, 2023, 65 (01) : 67 - 77Dangouloff, Tamara论文数: 0 引用数: 0 h-index: 0机构: Univ Hosp Liege, Dept Paediat, Reference Ctr Neuromuscular Dis, Div Child Neurol, Liege, Belgium Univ Liege, Liege, Belgium Univ Hosp Liege, Dept Paediat, Reference Ctr Neuromuscular Dis, Div Child Neurol, Liege, Belgium论文数: 引用数: h-index:机构:Deconinck, Nicolas论文数: 0 引用数: 0 h-index: 0机构: Univ Libre Bruxelles, Hop Univ Enfants Reine Fabiola, Neuromuscular Reference Ctr, Brussels, Belgium Univ Libre Bruxelles, Hop Univ Enfants Reine Fabiola, Paediat Neurol Dept, Brussels, Belgium Univ Hosp Liege, Dept Paediat, Reference Ctr Neuromuscular Dis, Div Child Neurol, Liege, BelgiumD'Amico, Adele论文数: 0 引用数: 0 h-index: 0机构: Bambino Gesu Pediat Hosp, Ist Ricovero & Cura Carattere Sci, Dept Neurosci, Unit Neuromuscular & Neurodegenerat Disorders, Rome, Italy Univ Hosp Liege, Dept Paediat, Reference Ctr Neuromuscular Dis, Div Child Neurol, Liege, BelgiumSeferian, Andreea M.论文数: 0 引用数: 0 h-index: 0机构: GH, Inst Myol, Paris, France Univ Hosp Liege, Dept Paediat, Reference Ctr Neuromuscular Dis, Div Child Neurol, Liege, BelgiumBoemer, Francois论文数: 0 引用数: 0 h-index: 0机构: Univ Liege, Dept Human Genet, Biochem Genet Lab, CHU Liege, Liege, Belgium Univ Hosp Liege, Dept Paediat, Reference Ctr Neuromuscular Dis, Div Child Neurol, Liege, BelgiumServais, Laurent论文数: 0 引用数: 0 h-index: 0机构: Univ Hosp Liege, Dept Paediat, Reference Ctr Neuromuscular Dis, Div Child Neurol, Liege, Belgium Univ Liege, Liege, Belgium Univ Oxford, Muscular Dystrophy UK Neuromuscular Ctr, Dept Paediat, Oxford, England Univ Hosp Liege, Dept Paediat, Reference Ctr Neuromuscular Dis, Div Child Neurol, Liege, Belgium