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- [1] A Randomized Phase 1 Safety, Pharmacokinetic and Pharmacodynamic Study of the Novel Myostatin Inhibitor Apitegromab (SRK-015): A Potential Treatment for Spinal Muscular Atrophy[J]. ADVANCES IN THERAPY, 2021, 38 (06) : 3203 - 3222Barrett, Doreen论文数: 0 引用数: 0 h-index: 0机构: Scholar Rock Inc, 301 Binney St,3rd Floor, Cambridge, MA 02142 USA Scholar Rock Inc, 301 Binney St,3rd Floor, Cambridge, MA 02142 USABilic, Sanela论文数: 0 引用数: 0 h-index: 0机构: Vanadro LLC, Urbandale, IA USA Scholar Rock Inc, 301 Binney St,3rd Floor, Cambridge, MA 02142 USAChyung, Yung论文数: 0 引用数: 0 h-index: 0机构: Scholar Rock Inc, 301 Binney St,3rd Floor, Cambridge, MA 02142 USA Scholar Rock Inc, 301 Binney St,3rd Floor, Cambridge, MA 02142 USACote, Shaun M.论文数: 0 引用数: 0 h-index: 0机构: Scholar Rock Inc, 301 Binney St,3rd Floor, Cambridge, MA 02142 USA Scholar Rock Inc, 301 Binney St,3rd Floor, Cambridge, MA 02142 USAIarrobino, Ryan论文数: 0 引用数: 0 h-index: 0机构: Scholar Rock Inc, 301 Binney St,3rd Floor, Cambridge, MA 02142 USA Scholar Rock Inc, 301 Binney St,3rd Floor, Cambridge, MA 02142 USAKacena, Katherine论文数: 0 引用数: 0 h-index: 0机构: Katherine Kacena Consulting, Natick, MA USA Scholar Rock Inc, 301 Binney St,3rd Floor, Cambridge, MA 02142 USAKalra, Ashish论文数: 0 引用数: 0 h-index: 0机构: Scholar Rock Inc, 301 Binney St,3rd Floor, Cambridge, MA 02142 USA Scholar Rock Inc, 301 Binney St,3rd Floor, Cambridge, MA 02142 USALong, Kimberly论文数: 0 引用数: 0 h-index: 0机构: Scholar Rock Inc, 301 Binney St,3rd Floor, Cambridge, MA 02142 USA Scholar Rock Inc, 301 Binney St,3rd Floor, Cambridge, MA 02142 USANomikos, George论文数: 0 引用数: 0 h-index: 0机构: Scholar Rock Inc, 301 Binney St,3rd Floor, Cambridge, MA 02142 USA Scholar Rock Inc, 301 Binney St,3rd Floor, Cambridge, MA 02142 USAPlace, Amy论文数: 0 引用数: 0 h-index: 0机构: Scholar Rock Inc, 301 Binney St,3rd Floor, Cambridge, MA 02142 USA Scholar Rock Inc, 301 Binney St,3rd Floor, Cambridge, MA 02142 USAStill, James Gordon论文数: 0 引用数: 0 h-index: 0机构: Clin Dev Solut LLC, Raleigh, NC USA Scholar Rock Inc, 301 Binney St,3rd Floor, Cambridge, MA 02142 USAVrishabhendra, Leela论文数: 0 引用数: 0 h-index: 0机构: Medpace Clin Pharmacol, Cincinnati, OH USA Scholar Rock Inc, 301 Binney St,3rd Floor, Cambridge, MA 02142 USA
- [2] 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
- [3] Integrating newborn screening for spinal muscular atrophy into health care systems: an Australian pilot programme[J]. DEVELOPMENTAL MEDICINE AND CHILD NEUROLOGY, 2022, 64 (05) : 625 - 632D'Silva, Arlene M.论文数: 0 引用数: 0 h-index: 0机构: Sydney Childrens Hosp Network, Dept Neurol, Sydney, NSW, Australia Univ New South Wales, Univ New South Wales Med & Hlth, Sch Womens & Childrens Hlth, Sydney, NSW, Australia Sydney Childrens Hosp Network, Dept Neurol, Sydney, NSW, AustraliaKariyawasam, Didu S. T.论文数: 0 引用数: 0 h-index: 0机构: Sydney Childrens Hosp Network, Dept Neurol, Sydney, NSW, Australia Univ New South Wales, Univ New South Wales Med & Hlth, Sch Womens & Childrens Hlth, Sydney, NSW, Australia Sydney Childrens Hosp Network, Dept Neurol, Sydney, NSW, AustraliaBest, Stephanie论文数: 0 引用数: 0 h-index: 0机构: Macquarie Univ, Australian Inst Hlth Innovat, Sydney, NSW, Australia Murdoch Childrens Res Inst, Australian Genom, Melbourne, Vic, Australia Sydney Childrens Hosp Network, Dept Neurol, Sydney, NSW, AustraliaWiley, Veronica论文数: 0 引用数: 0 h-index: 0机构: Childrens Hosp Westmead, NSW Newborn Screening Programme, Westmead, NSW, Australia Univ Sydney, Fac Med & Hlth, Sydney Med Sch, Discipline Child & Adolescent Hlth, Sydney, NSW, Australia Sydney Childrens Hosp Network, Dept Neurol, Sydney, NSW, AustraliaFarrar, Michelle A.论文数: 0 引用数: 0 h-index: 0机构: Sydney Childrens Hosp Network, Dept Neurol, Sydney, NSW, Australia Univ New South Wales, Univ New South Wales Med & Hlth, Sch Womens & Childrens Hlth, Sydney, NSW, Australia Sydney Childrens Hosp Network, Dept Neurol, Sydney, NSW, Australia
- [4] Nusinersen initiated in infants during the presymptomatic stage of spinal muscular atrophy: Interim efficacy and safety results from the Phase 2 NURTURE study[J]. NEUROMUSCULAR DISORDERS, 2019, 29 (11) : 842 - 856De Vivo, Darryl C.论文数: 0 引用数: 0 h-index: 0机构: Columbia Univ, Dept Neurol & Pediat, Irving Med Ctr, New York, NY 10032 USA Columbia Univ, Dept Neurol & Pediat, Irving Med Ctr, New York, NY 10032 USABertini, Enrico论文数: 0 引用数: 0 h-index: 0机构: IRCCS, Unit Neuromuscular & Neurodegenerat Disorders, Postgrad Bambino Gesu Childrens Res Hosp, Rome, Italy Columbia Univ, Dept Neurol & Pediat, Irving Med Ctr, New York, NY 10032 USASwoboda, Kathryn J.论文数: 0 引用数: 0 h-index: 0机构: Massachusetts Gen Hosp, Dept Neurol, Ctr Genom Med, Boston, MA 02114 USA Columbia Univ, Dept Neurol & Pediat, Irving Med Ctr, New York, NY 10032 USAHwu, Wuh-Liang论文数: 0 引用数: 0 h-index: 0机构: Natl Taiwan Univ Hosp, Dept Med Genet & Pediat, Taipei, Taiwan Columbia Univ, Dept Neurol & Pediat, Irving Med Ctr, New York, NY 10032 USACrawford, Thomas O.论文数: 0 引用数: 0 h-index: 0机构: Johns Hopkins Univ, Sch Med, Dept Neurol, Baltimore, MD 21205 USA Columbia Univ, Dept Neurol & Pediat, Irving Med Ctr, New York, NY 10032 USAFinkel, Richard S.论文数: 0 引用数: 0 h-index: 0机构: Nemours Childrens Hosp, Dept Pediat, Div Neurol, Orlando, FL USA Columbia Univ, Dept Neurol & Pediat, Irving Med Ctr, New York, NY 10032 USAKirschner, Janbernd论文数: 0 引用数: 0 h-index: 0机构: Univ Freiburg, Fac Med, Dept Neuropediat & Muscle Disorders, Freiburg, Germany Univ Med Hosp, Dept Neuropediat, Bonn, Germany Columbia Univ, Dept Neurol & Pediat, Irving Med Ctr, New York, NY 10032 USAKuntz, Nancy L.论文数: 0 引用数: 0 h-index: 0机构: Ann & Robert H Lurie Childrens Hosp Chicago, Chicago, IL 60611 USA Columbia Univ, Dept Neurol & Pediat, Irving Med Ctr, New York, NY 10032 USAParsons, Julie A.论文数: 0 引用数: 0 h-index: 0机构: Univ Colorado, Childrens Hosp Colorado, Sch Med, Aurora, CO USA Columbia Univ, Dept Neurol & Pediat, Irving Med Ctr, New York, NY 10032 USARyan, Monique M.论文数: 0 引用数: 0 h-index: 0机构: Univ Melbourne, Royal Childrens Hosp, Murdoch Childrens Res Inst, Melbourne, Vic, Australia Columbia Univ, Dept Neurol & Pediat, Irving Med Ctr, New York, NY 10032 USAButterfield, Russell J.论文数: 0 引用数: 0 h-index: 0机构: Univ Utah, Dept Pediat & Neurol, Salt Lake City, UT USA Columbia Univ, Dept Neurol & Pediat, Irving Med Ctr, New York, NY 10032 USATopaloglu, Haluk论文数: 0 引用数: 0 h-index: 0机构: Hacettepe Univ, Dept Pediat Neurol, Ankara, Turkey Columbia Univ, Dept Neurol & Pediat, Irving Med Ctr, New York, NY 10032 USABen-Omran, Tawfeg论文数: 0 引用数: 0 h-index: 0机构: Qatar Fdn, Dept Pediat, Sidra Med, Doha, Qatar Hamad Med Corp, Div Clin & Metab Genet, Dept Pediat, Doha, Qatar Columbia Univ, Dept Neurol & Pediat, Irving Med Ctr, New York, NY 10032 USASansone, Valeria A.论文数: 0 引用数: 0 h-index: 0机构: NEMO Clin Ctr NEuroMuscular Omniserv, Milan, Italy Univ Milan, Dept Biomed Sci Hlth, Milan, Italy Columbia Univ, Dept Neurol & Pediat, Irving Med Ctr, New York, NY 10032 USAJong, Yuh-Jyh论文数: 0 引用数: 0 h-index: 0机构: Kaohsiung Med Univ, Kaohsiung Med Univ Hosp, Dept Pediat & Lab Med, Coll Med, Kaohsiung, Taiwan Columbia Univ, Dept Neurol & Pediat, Irving Med Ctr, New York, NY 10032 USAShu, Francy论文数: 0 引用数: 0 h-index: 0机构: Univ Calif Los Angeles, David Geffen Sch Med, Dept Neurol, Los Angeles, CA 90095 USA Columbia Univ, Dept Neurol & Pediat, Irving Med Ctr, New York, NY 10032 USAStaropoli, John F.论文数: 0 引用数: 0 h-index: 0机构: Biogen, Cambridge, MA USA Columbia Univ, Dept Neurol & Pediat, Irving Med Ctr, New York, NY 10032 USAKerr, Douglas论文数: 0 引用数: 0 h-index: 0机构: Biogen, Cambridge, MA USA Columbia Univ, Dept Neurol & Pediat, Irving Med Ctr, New York, NY 10032 USASandrock, Alfred W.论文数: 0 引用数: 0 h-index: 0机构: Biogen, Cambridge, MA USA Columbia Univ, Dept Neurol & Pediat, Irving Med Ctr, New York, NY 10032 USAStebbins, Christopher论文数: 0 引用数: 0 h-index: 0机构: Biogen, Cambridge, MA USA Columbia Univ, Dept Neurol & Pediat, Irving Med Ctr, New York, NY 10032 USAPetrillo, Marco论文数: 0 引用数: 0 h-index: 0机构: Biogen, Cambridge, MA USA Columbia Univ, Dept Neurol & Pediat, Irving Med Ctr, New York, NY 10032 USABraley, Gabriel论文数: 0 引用数: 0 h-index: 0机构: Biogen, Cambridge, MA USA Columbia Univ, Dept Neurol & Pediat, Irving Med Ctr, New York, NY 10032 USAJohnson, Kristina论文数: 0 引用数: 0 h-index: 0机构: Biogen, Cambridge, MA USA Columbia Univ, Dept Neurol & Pediat, Irving Med Ctr, New York, NY 10032 USAFoster, Richard论文数: 0 引用数: 0 h-index: 0机构: Biogen, Maidenhead, Berks, England Columbia Univ, Dept Neurol & Pediat, Irving Med Ctr, New York, NY 10032 USAGheuens, Sarah论文数: 0 引用数: 0 h-index: 0机构: Biogen, Cambridge, MA USA Columbia Univ, Dept Neurol & Pediat, Irving Med Ctr, New York, NY 10032 USABhan, Ishir论文数: 0 引用数: 0 h-index: 0机构: Biogen, Cambridge, MA USA Columbia Univ, Dept Neurol & Pediat, Irving Med Ctr, New York, NY 10032 USAReyna, Sandra P.论文数: 0 引用数: 0 h-index: 0机构: Biogen, Cambridge, MA USA Columbia Univ, Dept Neurol & Pediat, Irving Med Ctr, New York, NY 10032 USAFradette, Stephanie论文数: 0 引用数: 0 h-index: 0机构: Biogen, Cambridge, MA USA Columbia Univ, Dept Neurol & Pediat, Irving Med Ctr, New York, NY 10032 USAFarwell, Wildon论文数: 0 引用数: 0 h-index: 0机构: Biogen, Cambridge, MA USA Columbia Univ, Dept Neurol & Pediat, Irving Med Ctr, New York, NY 10032 USA
- [5] Presymptomatic spinal muscular atrophy: a cautionary approach to the proposed new terminology[J]. BRAIN, 2023, 146 (09) : E65 - E66Farrar, Michelle A.论文数: 0 引用数: 0 h-index: 0机构: UNSW, Discipline Paediat & Child Hlth, UNSW Med & Hlth, Sydney, NSW 2052, Australia Sydney Childrens Hosp Network, Dept Neurol, Sydney, NSW 2031, Australia Sydney Childrens Hosp Network, Level 8,Bright Alliance Bldg,Avoca St, Randwick, NSW 2031, Australia UNSW, Discipline Paediat & Child Hlth, UNSW Med & Hlth, Sydney, NSW 2052, AustraliaKiernan, Matthew C.论文数: 0 引用数: 0 h-index: 0机构: Univ Sydney, Brain & Mind Ctr, Sydney, NSW 2050, Australia Royal Prince Alfred Hosp, Dept Neurol, Sydney, NSW 2050, Australia UNSW, Discipline Paediat & Child Hlth, UNSW Med & Hlth, Sydney, NSW 2052, AustraliaKariyawasam, Didu S.论文数: 0 引用数: 0 h-index: 0机构: UNSW, Discipline Paediat & Child Hlth, UNSW Med & Hlth, Sydney, NSW 2052, Australia Sydney Childrens Hosp Network, Dept Neurol, Sydney, NSW 2031, Australia UNSW, Discipline Paediat & Child Hlth, UNSW Med & Hlth, Sydney, NSW 2052, Australia
- [6] Newborn screening for spinal muscular atrophy in Australia: a non-randomised cohort study[J]. LANCET CHILD & ADOLESCENT HEALTH, 2023, 7 (03) : 159 - 170Kariyawasam, Didu S.论文数: 0 引用数: 0 h-index: 0机构: Sydney Childrens Hosp Network, Dept Neurol, Sydney, NSW, Australia Univ New South Wales, Sch Clin Med Med & Hlth, Discipline Paediat & Child Hlth, Sydney, NSW, Australia Sydney Childrens Hosp Network, Dept Neurol, Sydney, NSW 2035, Australia Sydney Childrens Hosp Network, Dept Neurol, Sydney, NSW, AustraliaD'Silva, Arlene M.论文数: 0 引用数: 0 h-index: 0机构: Univ New South Wales, Sch Clin Med Med & Hlth, Discipline Paediat & Child Hlth, Sydney, NSW, Australia Sydney Childrens Hosp Network, Dept Neurol, Sydney, NSW, AustraliaSampaio, Hugo论文数: 0 引用数: 0 h-index: 0机构: Sydney Childrens Hosp Network, Dept Neurol, Sydney, NSW, Australia Sydney Childrens Hosp Network, Dept Neurol, Sydney, NSW, AustraliaBriggs, Nancy论文数: 0 引用数: 0 h-index: 0机构: Univ New South Wales, Mark Wainwright Analyt Ctr, Sydney, NSW, Australia Sydney Childrens Hosp Network, Dept Neurol, Sydney, NSW, AustraliaHerbert, Karen论文数: 0 引用数: 0 h-index: 0机构: Sydney Childrens Hosp Network, Dept Physiotherapy, Sydney, NSW, Australia Sydney Childrens Hosp Network, Dept Neurol, Sydney, NSW, AustraliaWiley, Veronica论文数: 0 引用数: 0 h-index: 0机构: Childrens Hosp Westmead, New South Wales Newborn Screening Program, Westmead, NSW, Australia Univ Sydney, Fac Med & Hlth, Sydney, NSW, Australia Sydney Childrens Hosp Network, Dept Neurol, Sydney, NSW, AustraliaFarrar, Michelle A.论文数: 0 引用数: 0 h-index: 0机构: Sydney Childrens Hosp Network, Dept Neurol, Sydney, NSW, Australia Univ New South Wales, Sch Clin Med Med & Hlth, Discipline Paediat & Child Hlth, Sydney, NSW, Australia Sydney Childrens Hosp Network, Dept Neurol, Sydney, NSW, Australia
- [7] Biomarkers and the Development of a Personalized Medicine Approach in Spinal Muscular Atrophy[J]. FRONTIERS IN NEUROLOGY, 2019, 10Kariyawasam, Didu S. T.论文数: 0 引用数: 0 h-index: 0机构: Sydney Childrens Hosp, Dept Neurol, Sydney, NSW, Australia Univ New South Wales, Univ New South Wales Med, Sch Womens & Childrens Hlth, Sydney, NSW, Australia Sydney Childrens Hosp, Dept Neurol, Sydney, NSW, AustraliaD'Silva, Arlene论文数: 0 引用数: 0 h-index: 0机构: Univ New South Wales, Univ New South Wales Med, Sch Womens & Childrens Hlth, Sydney, NSW, Australia Sydney Childrens Hosp, Dept Neurol, Sydney, NSW, AustraliaLin, Cindy论文数: 0 引用数: 0 h-index: 0机构: Univ Sydney, Brain & Mind Ctr, Dept Neurophysiol, Sydney, NSW, Australia Sydney Childrens Hosp, Dept Neurol, Sydney, NSW, AustraliaRyan, Monique M.论文数: 0 引用数: 0 h-index: 0机构: Univ Melbourne, Royal Childrens Hosp, Dept Neurol, Murdoch Childrens Res Inst, Melbourne, Vic, Australia Sydney Childrens Hosp, Dept Neurol, Sydney, NSW, AustraliaFarrar, Michelle A.论文数: 0 引用数: 0 h-index: 0机构: Sydney Childrens Hosp, Dept Neurol, Sydney, NSW, Australia Univ New South Wales, Univ New South Wales Med, Sch Womens & Childrens Hlth, Sydney, NSW, Australia Sydney Childrens Hosp, Dept Neurol, Sydney, NSW, Australia
- [8] Yeo CJJ, 2024, LANCET NEUROL, V23, P205, DOI 10.1016/S1474-4422(23)00419-2