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- [41] Expert recommendations and clinical considerations in the use of onasemnogene abeparvovec gene therapy for spinal muscular atrophyMUSCLE & NERVE, 2021, 64 (04) : 413 - 427Kichula, Elizabeth A.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 USAProud, Crystal M.论文数: 0 引用数: 0 h-index: 0机构: Childrens Hosp Kings Daughters, Norfolk, VA USA Childrens Hosp Philadelphia, Philadelphia, PA 19104 USAFarrar, Michelle A.论文数: 0 引用数: 0 h-index: 0机构: Univ New South Wales Sydney, UNSW Med, Sch Womens & Childrens Hlth, Sydney, NSW, Australia Sydney Childrens Hosp Network, Sydney, NSW, Australia Childrens Hosp Philadelphia, Philadelphia, PA 19104 USAKwon, Jennifer M.论文数: 0 引用数: 0 h-index: 0机构: Univ Wisconsin, Sch Med & Publ Hlth, Madison, WI USA Childrens Hosp Philadelphia, Philadelphia, PA 19104 USASaito, Kayoko论文数: 0 引用数: 0 h-index: 0机构: Tokyo Womens Med Univ, Inst Med Genet, Tokyo, Japan Childrens Hosp Philadelphia, Philadelphia, PA 19104 USADesguerre, Isabelle论文数: 0 引用数: 0 h-index: 0机构: Univ Paris, Necker Enfants Malades Hosp, AP HP, Paris, France Childrens Hosp Philadelphia, Philadelphia, PA 19104 USAMcMillan, Hugh J.论文数: 0 引用数: 0 h-index: 0机构: Univ Ottawa, Childrens Hosp Eastern Ontario, 401 Smyth Rd, Ottawa, ON K1H 8L1, Canada Childrens Hosp Philadelphia, Philadelphia, PA 19104 USA
- [42] Onasemnogene Abeparvovec for Presymptomatic Infants with Spinal Muscular Atrophy and Two Copies of SMN2NEUROLOGY, 2022, 98 (18)Strauss, Kevin论文数: 0 引用数: 0 h-index: 0机构: Clin Special Children, Strasburg, PA USA Penn Med, Lancaster Gen Hosp, Lancaster, PA USA Univ Massachusetts, Dept Pediat, Sch Med, Amherst, MA USA Univ Massachusetts, Dept Mol Cell & Canc Biol, Sch Med, Amherst, MA USA Clin Special Children, Strasburg, PA USAMuntoni, Francesco论文数: 0 引用数: 0 h-index: 0机构: UCL Inst Child Hlth, London, England Great Ormond St Hosp Biomed Res Ctr, Natl Inst Hlth Res, London, England Clin Special Children, Strasburg, PA USAFarrar, Michelle论文数: 0 引用数: 0 h-index: 0机构: Sydney Childrens Hosp Network, Dept Neurol, Sydney, NSW, Australia UNSW Sydney, Sydney, NSW, Australia Clin Special Children, Strasburg, PA USASaito, Kayoko论文数: 0 引用数: 0 h-index: 0机构: Tokyo Womens Med Univ, Inst Med Genet, Tokyo, Japan Clin Special Children, Strasburg, PA USAMendell, Jerry论文数: 0 引用数: 0 h-index: 0机构: Nationwide Childrens Hosp, Ctr Gene Therapy, Columbus, OH USA Ohio State Univ, Dept Pediat, Columbus, OH 43210 USA Ohio State Univ, Dept Neurol, Columbus, OH 43210 USA Clin Special Children, Strasburg, PA USAServals, Laurent论文数: 0 引用数: 0 h-index: 0机构: Univ Oxford, MDUK Oxford Neuromuscular Ctr, Dept Paediat, Oxford, England Clin Special Children, Strasburg, PA USAMcMillan, Hugh论文数: 0 引用数: 0 h-index: 0机构: Childrens Hosp Eastern Ontario, Dept Pediat, Ottawa, ON, Canada Clin Special Children, Strasburg, PA USAFinkel, Richard论文数: 0 引用数: 0 h-index: 0机构: St Jude Childrens Res Hosp, Translat Neurosci Program, Memphis, TN USA Nemours Childrens Hosp, Dept Pediat, Orlando, FL USA Clin Special Children, Strasburg, PA USASwoboda, Kathryn论文数: 0 引用数: 0 h-index: 0机构: Massachusetts Gen Hosp, Dept Neurol, Boston, MA 02114 USA Clin Special Children, Strasburg, PA USAKwon, Jennifer论文数: 0 引用数: 0 h-index: 0机构: Univ Wisconsin, Dept Neurol, Sch Med & Publ Hlth, Madison, WI USA Clin Special Children, Strasburg, PA USAZaidman, Craig论文数: 0 引用数: 0 h-index: 0机构: Univ Washington, Sch Med, Seattle, WA USA Clin Special Children, Strasburg, PA USAChiriboga, Claudia论文数: 0 引用数: 0 h-index: 0机构: Columbia Univ, Div Pediat Neurol, Med Ctr, New York, NY USA Clin Special Children, Strasburg, PA USAIannaccone, Susan论文数: 0 引用数: 0 h-index: 0机构: Univ Texas Southwestern Med Ctr Dallas, Dept Pediat, Dallas, TX USA Clin Special Children, Strasburg, PA USAKrueger, Jena论文数: 0 引用数: 0 h-index: 0机构: Helen DeVos Childrens Hosp, Dept Neurol, Grand Rapids, MI USA Clin Special Children, Strasburg, PA USAParsons, Julie论文数: 0 引用数: 0 h-index: 0机构: Univ Colorado, Sch Med, Dept Pediat, Boulder, CO 80309 USA Clin Special Children, Strasburg, PA USAShieh, Perry论文数: 0 引用数: 0 h-index: 0机构: Univ Calif Los Angeles, Dept Neurol, David Geffen Sch Med, Los Angeles, CA USA Clin Special Children, Strasburg, PA USAKavanagh, Sarah论文数: 0 引用数: 0 h-index: 0机构: Novartis Gene Therapies Inc, Bannockburn, IL USA Clin Special Children, Strasburg, PA USAChand, Deepa论文数: 0 引用数: 0 h-index: 0机构: Univ Washington, Sch Med, Seattle, WA USA Novartis Gene Therapies Inc, Bannockburn, IL USA Clin Special Children, Strasburg, PA USATauscher-Wisniewski, Sitra论文数: 0 引用数: 0 h-index: 0机构: Novartis Gene Therapies Inc, Bannockburn, IL USA Clin Special Children, Strasburg, PA USAMcGill, Bryan论文数: 0 引用数: 0 h-index: 0机构: Novartis Gene Therapies Inc, Bannockburn, IL USA Clin Special Children, Strasburg, PA USAMacek, Thomas论文数: 0 引用数: 0 h-index: 0机构: Novartis Gene Therapies Inc, Bannockburn, IL USA Clin Special Children, Strasburg, PA USA
- [43] Long-term follow-up (LTFU) of onasemnogene abeparvovec gene therapy in spinal muscular atrophy (SMA)NEUROMUSCULAR DISORDERS, 2021, 31 : S132 - S133Mendell, J.论文数: 0 引用数: 0 h-index: 0机构: Nationwide Childrens Hosp, Columbus, OH USA Nationwide Childrens Hosp, Columbus, OH USAFinkel, R.论文数: 0 引用数: 0 h-index: 0机构: St Jude Childrens Res Hosp, 332 N Lauderdale St, Memphis, TN 38105 USA Nationwide Childrens Hosp, Columbus, OH USAMercuri, E.论文数: 0 引用数: 0 h-index: 0机构: Catholic Univ, Rome, Italy Nationwide Childrens Hosp, Columbus, OH USAStrauss, K.论文数: 0 引用数: 0 h-index: 0机构: Clin Special Children, Strasburg, PA USA Nationwide Childrens Hosp, Columbus, OH USADay, J.论文数: 0 引用数: 0 h-index: 0机构: Stanford Univ, Med Ctr, Palo Alto, CA 94304 USA Nationwide Childrens Hosp, Columbus, OH USAKleyn, A.论文数: 0 引用数: 0 h-index: 0机构: Novartis Gene Therapies, Bannockburn, IL USA Nationwide Childrens Hosp, Columbus, OH USAChand, D.论文数: 0 引用数: 0 h-index: 0机构: Novartis Gene Therapies, Bannockburn, IL USA Nationwide Childrens Hosp, Columbus, OH USATauscher-Wisniewski, S.论文数: 0 引用数: 0 h-index: 0机构: Novartis Gene Therapies, Bannockburn, IL USA Nationwide Childrens Hosp, Columbus, OH USAMeriggioli, M.论文数: 0 引用数: 0 h-index: 0机构: Novartis Gene Therapies, Bannockburn, IL USA Nationwide Childrens Hosp, Columbus, OH USA
- [44] Effectiveness and Safety of Onasemnogene Abeparvovec in Older Patients with Spinal Muscular Atrophy (SMA): Real-World Outcomes from the RESTORE RegistryNEUROLOGY, 2022, 98 (18)Servais, Laurent论文数: 0 引用数: 0 h-index: 0机构: Univ Oxford, MDUK Oxford Neuromuscular Ctr, Dept Pediat, Oxford, England Univ Oxford, MDUK Oxford Neuromuscular Ctr, Dept Pediat, Oxford, EnglandDe Vivo, Darryl论文数: 0 引用数: 0 h-index: 0机构: Columbia Univ, Dept Neurol, Irving Med Ctr, New York, NY 10027 USA Columbia Univ, Dept Pediat, Irving Med Ctr, New York, NY 10027 USA Univ Oxford, MDUK Oxford Neuromuscular Ctr, Dept Pediat, Oxford, EnglandKirschner, Janbernd论文数: 0 引用数: 0 h-index: 0机构: Univ Med Ctr Freiburg, Clin Neuropediat & Muscle Dis, Freiburg, Germany Univ Oxford, MDUK Oxford Neuromuscular Ctr, Dept Pediat, Oxford, EnglandMercuri, Eugenio论文数: 0 引用数: 0 h-index: 0机构: Catholic Univ, Dept Paediat Neurol, Washington, DC USA Catholic Univ, Nemo Clin Ctr, Washington, DC USA Univ Oxford, MDUK Oxford Neuromuscular Ctr, Dept Pediat, Oxford, EnglandMuntoni, Francesco论文数: 0 引用数: 0 h-index: 0机构: UCL Inst Child Hlth, Dubowitz Neuromuscular Ctr, London, England Great Ormond St Hosp Biomed Res Ctr, Natl Inst Hlth Res, London, England Univ Oxford, MDUK Oxford Neuromuscular Ctr, Dept Pediat, Oxford, EnglandTizzano, 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 Oxford, MDUK Oxford Neuromuscular Ctr, Dept Pediat, Oxford, EnglandQuijano-Roy, Susana论文数: 0 引用数: 0 h-index: 0机构: UVSQ Paris Saclay, Garches Neuromuscular Reference Ctr GNMH, Raymond Poincare Univ Hosp, AP HP, Paris, France Univ Oxford, MDUK Oxford Neuromuscular Ctr, Dept Pediat, Oxford, EnglandSaito, Kayoko论文数: 0 引用数: 0 h-index: 0机构: Tokyo Womens Med Univ, Inst Med Genet, Tokyo, Japan Univ Oxford, MDUK Oxford Neuromuscular Ctr, Dept Pediat, Oxford, EnglandMenier, Melissa论文数: 0 引用数: 0 h-index: 0机构: Atlas Clar LLC, San Francisco, CA USA Univ Oxford, MDUK Oxford Neuromuscular Ctr, Dept Pediat, Oxford, EnglandLaMarca, Nicole论文数: 0 引用数: 0 h-index: 0机构: Novartis Gene Therapies Inc, Dallas, TX USA Univ Oxford, MDUK Oxford Neuromuscular Ctr, Dept Pediat, Oxford, EnglandAnderson, Fred论文数: 0 引用数: 0 h-index: 0机构: Univ Massachusetts, Ctr Outcomes Res, Med Sch, Amherst, MA 01003 USA Univ Oxford, MDUK Oxford Neuromuscular Ctr, Dept Pediat, Oxford, EnglandDabbous, Omar论文数: 0 引用数: 0 h-index: 0机构: Novartis Gene Therapies Inc, Dallas, TX USA Univ Oxford, MDUK Oxford Neuromuscular Ctr, Dept Pediat, Oxford, EnglandFinkel, Richard论文数: 0 引用数: 0 h-index: 0机构: St Jude Childrens Res Hosp, New York, NY USA Univ Oxford, MDUK Oxford Neuromuscular Ctr, Dept Pediat, Oxford, England
- [45] Clinical features of spinal muscular atrophy (SMA) type 2ARCHIVES DE PEDIATRIE, 2020, 27 (07): : 18 - 22Cances, C.论文数: 0 引用数: 0 h-index: 0机构: Toulouse Univ Hosp, Neuropaediat Dept, AOC Atlantique Occitante Caraibe Reference Ctr Ne, Toulouse, France Toulouse Univ Hosp, Neuropaediat Dept, AOC Atlantique Occitante Caraibe Reference Ctr Ne, Toulouse, FranceRichelme, C.论文数: 0 引用数: 0 h-index: 0机构: Hop Pediat Nice CHU Lenval, CMR Neuromusculaire PACARARE, Nice, France Toulouse Univ Hosp, Neuropaediat Dept, AOC Atlantique Occitante Caraibe Reference Ctr Ne, Toulouse, FranceBarnerias, C.论文数: 0 引用数: 0 h-index: 0机构: CMR Neuromusculaire NEIDF, AP HP, Paris, France Toulouse Univ Hosp, Neuropaediat Dept, AOC Atlantique Occitante Caraibe Reference Ctr Ne, Toulouse, FranceEspil, C.论文数: 0 引用数: 0 h-index: 0机构: CHU Bordeaux, Hop Enfants, CMR Neuromusculaire AOC, Bordeaux, France Toulouse Univ Hosp, Neuropaediat Dept, AOC Atlantique Occitante Caraibe Reference Ctr Ne, Toulouse, France
- [46] Necrotizing Enterocolitis following Onasemnogene Abeparvovec for Spinal Muscular Atrophy: A Case SeriesJOURNAL OF PEDIATRICS, 2023, 260Gaillard, Jonathan论文数: 0 引用数: 0 h-index: 0机构: Univ Michigan, Dept Pediat, Div Neurol, Ann Arbor, MI 48109 USA Univ Michigan, Dept Pediat, Div Neurol, Ann Arbor, MI 48109 USAGu, Andrew Ran论文数: 0 引用数: 0 h-index: 0机构: Univ Michigan, Dept Pediat, Ann Arbor, MI USA Univ Michigan, Dept Pediat, Div Neurol, Ann Arbor, MI 48109 USAKnierbein, Erin E. Neil论文数: 0 引用数: 0 h-index: 0机构: Univ Michigan, Dept Pediat, Div Neurol, Ann Arbor, MI 48109 USA Univ Michigan, Dept Pediat, Div Neurol, Ann Arbor, MI 48109 USA
- [47] Thrombotic Microangiopathy Following Onasemnogene Abeparvovec for Spinal Muscular Atrophy: A Case SeriesJOURNAL OF PEDIATRICS, 2021, 231 : 265 - 268Chand, Deepa H.论文数: 0 引用数: 0 h-index: 0机构: Novartis Gene Therapies, Bannockburn, IL 60015 USA Washington Univ, Sch Med, St Louis, MO USA St Louis Childrens Hosp, St Louis, MO 63178 USA Novartis Gene Therapies, Bannockburn, IL 60015 USAZaidman, Craig论文数: 0 引用数: 0 h-index: 0机构: Washington Univ, Sch Med, St Louis, MO USA St Louis Childrens Hosp, St Louis, MO 63178 USA Novartis Gene Therapies, Bannockburn, IL 60015 USAArya, Kapil论文数: 0 引用数: 0 h-index: 0机构: Univ Arkansas Med Sci, Little Rock, AR 72205 USA Novartis Gene Therapies, Bannockburn, IL 60015 USAMillner, Rachel论文数: 0 引用数: 0 h-index: 0机构: Univ Arkansas Med Sci, Little Rock, AR 72205 USA Novartis Gene Therapies, Bannockburn, IL 60015 USAFarrar, Michelle A.论文数: 0 引用数: 0 h-index: 0机构: Sydney Childrens Hosp Randwick, Randwick, NSW, Australia Univ New South Wales, UNSW Sydney, Sch Womens & Childrens Hlth, UNSW Med, Sydney, NSW, Australia Novartis Gene Therapies, Bannockburn, IL 60015 USAMackie, Fiona E.论文数: 0 引用数: 0 h-index: 0机构: Sydney Childrens Hosp Randwick, Randwick, NSW, Australia Univ New South Wales, UNSW Sydney, Sch Womens & Childrens Hlth, UNSW Med, Sydney, NSW, Australia Novartis Gene Therapies, Bannockburn, IL 60015 USAGoedeker, Natalie L.论文数: 0 引用数: 0 h-index: 0机构: Washington Univ, Sch Med, St Louis, MO USA St Louis Childrens Hosp, St Louis, MO 63178 USA Novartis Gene Therapies, Bannockburn, IL 60015 USADharnidharka, Vikas R.论文数: 0 引用数: 0 h-index: 0机构: Washington Univ, Sch Med, St Louis, MO USA St Louis Childrens Hosp, St Louis, MO 63178 USA Novartis Gene Therapies, Bannockburn, IL 60015 USADandamudi, Raja论文数: 0 引用数: 0 h-index: 0机构: Washington Univ, Sch Med, St Louis, MO USA St Louis Childrens Hosp, St Louis, MO 63178 USA Novartis Gene Therapies, Bannockburn, IL 60015 USAReyna, Sandra P.论文数: 0 引用数: 0 h-index: 0机构: Novartis Gene Therapies, Bannockburn, IL 60015 USA Novartis Gene Therapies, Bannockburn, IL 60015 USA
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- [50] Expanding the Availability of Onasemnogene Abeparvovec to Older Patients: The Evolving Treatment Landscape for Spinal Muscular AtrophyPHARMACEUTICS, 2023, 15 (06)Rene, Charlotte A.论文数: 0 引用数: 0 h-index: 0机构: Ottawa Hosp Res Inst, Regenerat Med Program, Ottawa, ON K1H 8L6, Canada Univ Ottawa, Dept Biochem Microbiol & Immunol, Ottawa, ON K1H 8M5, Canada Univ Ottawa, Ctr Neuromuscular Dis, Ottawa, ON K1H 8M5, Canada Ottawa Hosp Res Inst, Regenerat Med Program, Ottawa, ON K1H 8L6, CanadaParks, Robin J.论文数: 0 引用数: 0 h-index: 0机构: Ottawa Hosp Res Inst, Regenerat Med Program, Ottawa, ON K1H 8L6, Canada Univ Ottawa, Dept Biochem Microbiol & Immunol, Ottawa, ON K1H 8M5, Canada Univ Ottawa, Ctr Neuromuscular Dis, Ottawa, ON K1H 8M5, Canada Ottawa Hosp, Dept Med, Ottawa, ON K1H 8L6, Canada Ottawa Hosp Res Inst, Regenerat Med Program, Ottawa, ON K1H 8L6, Canada