共 50 条
- [1] 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
- [2] Onasemnogene-abeparvovec administration to premature infants with spinal muscular atrophyANNALS OF CLINICAL AND TRANSLATIONAL NEUROLOGY, 2024, 11 (11): : 3042 - 3046Brown, Stephen M.论文数: 0 引用数: 0 h-index: 0机构: Johns Hopkins Univ, Sch Med, Dept Neurol, Baltimore, MD USA Johns Hopkins Univ, Sch Med, Dept Neurol, Baltimore, MD USAAjjarapu, Aparna S.论文数: 0 引用数: 0 h-index: 0机构: Univ Iowa, Carver Coll Med, Dept Pediat, 1317 Boyd Tower,200 Hawkins Dr, Iowa City, IA 52242 USA Johns Hopkins Univ, Sch Med, Dept Neurol, Baltimore, MD USARamachandra, Divya论文数: 0 引用数: 0 h-index: 0机构: Advocate Childrens Hosp, Dept Genet, Oak Lawn, IL USA Johns Hopkins Univ, Sch Med, Dept Neurol, Baltimore, MD USABlasco-Perez, Laura论文数: 0 引用数: 0 h-index: 0机构: Hosp Valle De Hebron, Dept Clin & Mol Genet, VHIR, Barcelona, Spain Hosp Valle De Hebron, Med Genet Grp, VHIR, Barcelona, Spain Johns Hopkins Univ, Sch Med, Dept Neurol, Baltimore, MD USACosta-Roger, Mar论文数: 0 引用数: 0 h-index: 0机构: Hosp Valle De Hebron, Dept Clin & Mol Genet, VHIR, Barcelona, Spain Hosp Valle De Hebron, Med Genet Grp, VHIR, Barcelona, Spain Johns Hopkins Univ, Sch Med, Dept Neurol, Baltimore, MD USATizzano, Eduardo F.论文数: 0 引用数: 0 h-index: 0机构: Hosp Valle De Hebron, Dept Clin & Mol Genet, VHIR, Barcelona, Spain Hosp Valle De Hebron, Med Genet Grp, VHIR, Barcelona, Spain Johns Hopkins Univ, Sch Med, Dept Neurol, Baltimore, MD USASumner, Charlotte J.论文数: 0 引用数: 0 h-index: 0机构: Johns Hopkins Univ, Sch Med, Dept Neurol, Baltimore, MD USA Johns Hopkins Univ, Sch Med, Dept Neurol, Baltimore, MD USAMathews, Katherine D.论文数: 0 引用数: 0 h-index: 0机构: Univ Iowa, Carver Coll Med, Dept Pediat, 1317 Boyd Tower,200 Hawkins Dr, Iowa City, IA 52242 USA Univ Iowa, Carver Coll Med, Dept Neurol, Iowa City, IA USA Johns Hopkins Univ, Sch Med, Dept Neurol, Baltimore, MD USA
- [3] Polysomnography findings in children with spinal muscular atrophy after onasemnogene-abeparvovecSLEEP MEDICINE, 2023, 101 : 234 - 237Leon-Astudillo, Carmen论文数: 0 引用数: 0 h-index: 0机构: Univ Florida, Dept Pediat, Div Pediat Pulmonol, Coll Med, Gainesville, FL USA Univ Florida, Dept Pediat, Div Pediat Pulmonol, Coll Med, 1600 SW Archer Rd, Gainesville, FL 32610 USA Univ Florida, Dept Pediat, Div Pediat Pulmonol, Coll Med, Gainesville, FL USAWagner, Mary论文数: 0 引用数: 0 h-index: 0机构: Univ Florida, Dept Pediat, Div Pediat Pulmonol, Coll Med, Gainesville, FL USA Univ Florida, Dept Pediat, Div Pediat Pulmonol, Coll Med, Gainesville, FL USASalabarria, Stephanie M.论文数: 0 引用数: 0 h-index: 0机构: Univ Florida, Coll Med, Dept Pediat, Gainesville, FL USA Univ Florida, Powell Gene Therapy Ctr, Gainesville, FL USA Univ Florida, Dept Pediat, Div Pediat Pulmonol, Coll Med, Gainesville, FL USALammers, Jenna论文数: 0 引用数: 0 h-index: 0机构: Univ Florida, Coll Med, Dept Pediat, Gainesville, FL USA Univ Florida, Powell Gene Therapy Ctr, Gainesville, FL USA Univ Florida, Dept Pediat, Div Pediat Pulmonol, Coll Med, Gainesville, FL USABerthy, Julie论文数: 0 引用数: 0 h-index: 0机构: Univ Florida, Coll Med, Dept Pediat, Gainesville, FL USA Univ Florida, Powell Gene Therapy Ctr, Gainesville, FL USA Univ Florida, Dept Pediat, Div Pediat Pulmonol, Coll Med, Gainesville, FL USAZingariello, Carla D.论文数: 0 引用数: 0 h-index: 0机构: Univ Florida, Dept Pediat, Div Pediat Neurol, Coll Med, Gainesville, FL USA Univ Florida, Dept Pediat, Div Pediat Pulmonol, Coll Med, Gainesville, FL USAByrne, Barry J.论文数: 0 引用数: 0 h-index: 0机构: Univ Florida, Coll Med, Dept Pediat, Gainesville, FL USA Univ Florida, Powell Gene Therapy Ctr, Gainesville, FL USA Univ Florida, Dept Pediat, Div Pediat Pulmonol, Coll Med, Gainesville, FL USASmith, Barbara K.论文数: 0 引用数: 0 h-index: 0机构: Univ Florida, Coll Med, Dept Pediat, Gainesville, FL USA Univ Florida, Dept Phys Therapy, Gainesville, FL USA Univ Florida, Dept Pediat, Div Pediat Pulmonol, Coll Med, Gainesville, FL USA
- [4] An evaluation of onasemnogene abeparvovec for spinal muscular atrophy (SMN1)EXPERT OPINION ON ORPHAN DRUGS, 2021, 9 (7-10): : 199 - 204Waldrop, Megan A.论文数: 0 引用数: 0 h-index: 0机构: Nationwide Childrens Hosp, Abigail Wexner Res Inst, Ctr Gene Therapy, Columbus, OH USA Ohio State Univ, Dept Pediat, Wexner Med Ctr, Columbus, OH 43210 USA Ohio State Univ, Dept Neurol, Wexner Med Ctr, Columbus, OH 43210 USA Nationwide Childrens Hosp, Abigail Wexner Res Inst, Ctr Gene Therapy, Columbus, OH USAConnolly, Anne M.论文数: 0 引用数: 0 h-index: 0机构: Nationwide Childrens Hosp, Abigail Wexner Res Inst, Ctr Gene Therapy, Columbus, OH USA Ohio State Univ, Dept Pediat, Wexner Med Ctr, Columbus, OH 43210 USA Ohio State Univ, Dept Neurol, Wexner Med Ctr, Columbus, OH 43210 USA Nationwide Childrens Hosp, Abigail Wexner Res Inst, Ctr Gene Therapy, Columbus, OH USAMendell, Jerry R.论文数: 0 引用数: 0 h-index: 0机构: Nationwide Childrens Hosp, Abigail Wexner Res Inst, Ctr Gene Therapy, Columbus, OH USA Ohio State Univ, Dept Pediat, Wexner Med Ctr, Columbus, OH 43210 USA Ohio State Univ, Dept Neurol, Wexner Med Ctr, Columbus, OH 43210 USA Nationwide Childrens Hosp, Abigail Wexner Res Inst, Ctr Gene Therapy, Columbus, OH USA
- [5] Onasemnogene Abeparvovec in Presymptomatic Spinal Muscular Atrophy (SMA): SPRINT Study Update in Children with Three 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 01003 USA Univ Massachusetts, Dept Mol Cell & Canc Biol, Sch Med, Amherst, MA 01003 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 Sick Children, Natl Inst Hlth Res, Biomed Res Ctr, 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, Res Inst, 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 USAServais, 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, 332 N Lauderdale St, Memphis, TN 38105 USA Nemours Childrens Hosp, Dept Pediat, Wilmington, DE 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, Sch Med & Publ Hlth, Dept Neurol, Madison, WI 53706 USA Clin Special Children, Strasburg, PA USAZaidman, Craig论文数: 0 引用数: 0 h-index: 0机构: Washington Univ, Sch Med, St Louis, MO USA Clin Special Children, Strasburg, PA USAChiriboga, Claudia论文数: 0 引用数: 0 h-index: 0机构: Columbia Univ, Med Ctr, Div Pediat Neurol, New York, NY 10027 USA Clin Special Children, Strasburg, PA USALannaccone, Susan论文数: 0 引用数: 0 h-index: 0机构: Univ Texas Southwestern Med Ctr, 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, David Geffen Sch Med, Dept Neurol, Los Angeles, CA 90024 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: Washington Univ, Sch Med, St Louis, MO 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
- [6] Outcomes in patients with spinal muscular atrophy and four or more SMN2 copies treated with onasemnogene abeparvovec: Findings from RESTORENEUROMUSCULAR DISORDERS, 2022, 32 : S59 - S59Finkel, 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 St Jude Childrens Res Hosp, 332 N Lauderdale St, Memphis, TN 38105 USABenguerba, K.论文数: 0 引用数: 0 h-index: 0机构: Novartis Gene Therapies Switzerland GmbH, Rotkreuz, Switzerland St Jude Childrens Res Hosp, 332 N Lauderdale St, Memphis, TN 38105 USAReid, A.论文数: 0 引用数: 0 h-index: 0机构: Novartis Global RWE Gene Therapies, London, England St Jude Childrens Res Hosp, 332 N Lauderdale St, Memphis, TN 38105 USARaju, D.论文数: 0 引用数: 0 h-index: 0机构: Novartis Gene Therapies Inc, Bannockburn, IL USA St Jude Childrens Res Hosp, 332 N Lauderdale St, Memphis, TN 38105 USAFaulkner, E.论文数: 0 引用数: 0 h-index: 0机构: Novartis Gene Therapies Inc, Bannockburn, IL USA St Jude Childrens Res Hosp, 332 N Lauderdale St, Memphis, TN 38105 USALaMarca, N.论文数: 0 引用数: 0 h-index: 0机构: Novartis Gene Therapies Inc, Bannockburn, IL USA St Jude Childrens Res Hosp, 332 N Lauderdale St, Memphis, TN 38105 USA论文数: 引用数: h-index:机构:
- [7] Outcomes for patients in the RESTORE registry with spinal muscular atrophy and four or more SMN2 gene copies treated with onasemnogene abeparvovecEUROPEAN JOURNAL OF PAEDIATRIC NEUROLOGY, 2024, 53 : 18 - 24Tizzano, Eduardo F.论文数: 0 引用数: 0 h-index: 0机构: Hosp Valle De Hebron, Dept Clin & Mol Genet, Passeig Vall dHebron 119-129,Horta Guinardo, Barcelona 08035, Spain Hosp Valle De Hebron, Dept Clin & Mol Genet, Passeig Vall dHebron 119-129,Horta Guinardo, Barcelona 08035, SpainQuijano-Roy, Susana论文数: 0 引用数: 0 h-index: 0机构: UVSQ Paris Saclay, Raymond Poincare Univ Hosp, APHP, Garches Neuromuscular Reference Ctr, 104 Bd Raymond Poincare, F-92380 Garches, France Hosp Valle De Hebron, Dept Clin & Mol Genet, Passeig Vall dHebron 119-129,Horta Guinardo, Barcelona 08035, Spain论文数: 引用数: h-index:机构:Parsons, A.论文数: 0 引用数: 0 h-index: 0机构: Hosp Valle De Hebron, Dept Clin & Mol Genet, Passeig Vall dHebron 119-129,Horta Guinardo, Barcelona 08035, SpainParsons, Julie A.论文数: 0 引用数: 0 h-index: 0机构: Univ Colorado, Childrens Hosp Colorado, Sch Med, 13001 East 17th Pl, Aurora, CO 80045 USA Hosp Valle De Hebron, Dept Clin & Mol Genet, Passeig Vall dHebron 119-129,Horta Guinardo, Barcelona 08035, SpainAharoni, Sharon论文数: 0 引用数: 0 h-index: 0机构: Inst Pediat Neurol, Schneider Childrens Med Ctr Israel, Kaplan St 14, Petah Tiqwa, Israel Tel Aviv Univ, Fac Med & Hlth Sci, Tel Aviv, Israel Hosp Valle De Hebron, Dept Clin & Mol Genet, Passeig Vall dHebron 119-129,Horta Guinardo, Barcelona 08035, Spain论文数: 引用数: h-index:机构:Finkel, Richard S.论文数: 0 引用数: 0 h-index: 0机构: St Jude Childrens Res Hosp, Ctr Prote & Metabol, 262 Danny Thomas Pl, Memphis, TN 38105 USA Hosp Valle De Hebron, Dept Clin & Mol Genet, Passeig Vall dHebron 119-129,Horta Guinardo, Barcelona 08035, Spain
- [8] Bulbar Function in Children with Two or Three SMN2 Copies Who Received Onasemnogene Abeparvovec Presymptomatically for Spinal Muscular AtrophyNEUROLOGY, 2023, 100 (17)论文数: 引用数: h-index:机构:论文数: 引用数: h-index:机构:Hurst-Davis, Rebecca论文数: 0 引用数: 0 h-index: 0机构: Primary Childrens Med Ctr, Salt Lake City, UT USA Univ Minnesota, Minneapolis, MN USAYoung, Sally Dunaway论文数: 0 引用数: 0 h-index: 0机构: Stanford Univ, Sch Med, Stanford, CA USA Univ Minnesota, Minneapolis, MN USABaranello, Giovanni论文数: 0 引用数: 0 h-index: 0机构: UCL Great Ormond St Inst Child Hlth, Biomed Res Ctr, Dubowitz Neuromuscular Ctr, Dev Neurosci Res & Teaching Dept, London, England NIHR Great Ormond St Hosp Biomed Res Ctr, London, England Great Ormond St Hosp NHS Fdn Trust, London, England Univ Minnesota, Minneapolis, MN USALavrov, Arseniy论文数: 0 引用数: 0 h-index: 0机构: Novartis Gene Therapies Inc, Basel, Switzerland Univ Minnesota, Minneapolis, MN USAO'Brien, Eamonn论文数: 0 引用数: 0 h-index: 0机构: Novartis, Basel, Switzerland Univ Minnesota, Minneapolis, MN USAWallach, Shiri论文数: 0 引用数: 0 h-index: 0机构: Novartis Gene Therapies Inc, Basel, Switzerland Univ Minnesota, Minneapolis, MN USALaMarca, Nicole论文数: 0 引用数: 0 h-index: 0机构: Novartis Gene Therapies Inc, Basel, Switzerland Univ Minnesota, Minneapolis, MN USAReyna, Sandra P.论文数: 0 引用数: 0 h-index: 0机构: Novartis Gene Therapies Inc, Basel, Switzerland Univ Minnesota, Minneapolis, MN USADarras, Basil论文数: 0 引用数: 0 h-index: 0机构: Harvard Med Sch, Boston Childrens Hosp, Boston, MA USA Univ Minnesota, Minneapolis, MN USA
- [9] Outcomes in Patients with Spinal Muscular Atrophy and Four or More SMN2 Copies Treated with Onasemnogene Abeparvovec: Findings from RESTORENEUROLOGY, 2023, 100 (17)Finkel, Richard论文数: 0 引用数: 0 h-index: 0机构: St Jude Childrens Res Hosp, Memphis, TN USA St Jude Childrens Res Hosp, Memphis, TN USABenguerba, Kamal论文数: 0 引用数: 0 h-index: 0机构: Novartis Gene Therapies Switzerland GmbH, Basel, Switzerland St Jude Childrens Res Hosp, Memphis, TN USAGehani, Manish论文数: 0 引用数: 0 h-index: 0机构: Novartis Healthcare Pvt Ltd, CONEXTS Real World Evidence, Basel, Switzerland St Jude Childrens Res Hosp, Memphis, TN USARaju, Dheeraj论文数: 0 引用数: 0 h-index: 0机构: Novartis Gene Therapies Inc, Basel, Switzerland St Jude Childrens Res Hosp, Memphis, TN USAFaulkner, Eric论文数: 0 引用数: 0 h-index: 0机构: Novartis Gene Therapies Inc, Basel, Switzerland St Jude Childrens Res Hosp, Memphis, TN USALaMarca, Nicole论文数: 0 引用数: 0 h-index: 0机构: Novartis Gene Therapies Inc, Basel, Switzerland St Jude Childrens Res Hosp, Memphis, TN USA论文数: 引用数: h-index:机构:
- [10] Outcomes in patients with spinal muscular atrophy (SMA) and four or more SMN2 copies treated with onasemnogene abeparvovec: findings from RESTORENEUROMUSCULAR DISORDERS, 2023, 33 : S133 - S133Finkel, R.论文数: 0 引用数: 0 h-index: 0机构: St Jude Childrens Res Hosp, Memphis, TN USA St Jude Childrens Res Hosp, Memphis, TN USABenguerba, K.论文数: 0 引用数: 0 h-index: 0机构: Novartis Gene Therapies Switzerland GmbH, Rotkreuz, Switzerland St Jude Childrens Res Hosp, Memphis, TN USAGehani, M.论文数: 0 引用数: 0 h-index: 0机构: Novartis Healthcare Pvt Ltd, CONEXTS Real World Evidence, Hyderabad, India St Jude Childrens Res Hosp, Memphis, TN USARaju, D.论文数: 0 引用数: 0 h-index: 0机构: Novartis Gene Therapies Inc, Bannockburn, IL USA St Jude Childrens Res Hosp, Memphis, TN USAFaulkner, E.论文数: 0 引用数: 0 h-index: 0机构: Novartis Gene Therapies Inc, Bannockburn, IL USA St Jude Childrens Res Hosp, Memphis, TN USALaMarca, N.论文数: 0 引用数: 0 h-index: 0机构: Novartis Gene Therapies Inc, Bannockburn, IL USA St Jude Childrens Res Hosp, Memphis, TN USAServais, L.论文数: 0 引用数: 0 h-index: 0机构: Univ Oxford, Dept Paediat, MDUK Oxford Neuromusc Ctr, Oxford, England St Jude Childrens Res Hosp, Memphis, TN USA