The Use of Onasemnogene-abeparvovec at 33 weeks' gestation in Spinal Muscular Atrophy with One Copy of SMN2

被引:1
|
作者
Ajjarapu, Aparna [1 ]
Ramachandra, Divya [2 ]
Mathews, Katherine [1 ]
机构
[1] Univ Iowa, Carver Coll Med, Dept Neurol, Iowa City, IA USA
[2] Advocate Childrens Hosp, Dept Genet, Park Ridge, IL USA
关键词
D O I
10.1212/WNL.0000000000203032
中图分类号
R74 [神经病学与精神病学];
学科分类号
摘要
P2-8.013
引用
收藏
页数:3
相关论文
共 50 条
  • [1] Onasemnogene Abeparvovec for Presymptomatic Infants with Spinal Muscular Atrophy and Two Copies of SMN2
    Strauss, Kevin
    Muntoni, Francesco
    Farrar, Michelle
    Saito, Kayoko
    Mendell, Jerry
    Servals, Laurent
    McMillan, Hugh
    Finkel, Richard
    Swoboda, Kathryn
    Kwon, Jennifer
    Zaidman, Craig
    Chiriboga, Claudia
    Iannaccone, Susan
    Krueger, Jena
    Parsons, Julie
    Shieh, Perry
    Kavanagh, Sarah
    Chand, Deepa
    Tauscher-Wisniewski, Sitra
    McGill, Bryan
    Macek, Thomas
    NEUROLOGY, 2022, 98 (18)
  • [2] Onasemnogene-abeparvovec administration to premature infants with spinal muscular atrophy
    Brown, Stephen M.
    Ajjarapu, Aparna S.
    Ramachandra, Divya
    Blasco-Perez, Laura
    Costa-Roger, Mar
    Tizzano, Eduardo F.
    Sumner, Charlotte J.
    Mathews, Katherine D.
    ANNALS OF CLINICAL AND TRANSLATIONAL NEUROLOGY, 2024, 11 (11): : 3042 - 3046
  • [3] Polysomnography findings in children with spinal muscular atrophy after onasemnogene-abeparvovec
    Leon-Astudillo, Carmen
    Wagner, Mary
    Salabarria, Stephanie M.
    Lammers, Jenna
    Berthy, Julie
    Zingariello, Carla D.
    Byrne, Barry J.
    Smith, Barbara K.
    SLEEP MEDICINE, 2023, 101 : 234 - 237
  • [4] An evaluation of onasemnogene abeparvovec for spinal muscular atrophy (SMN1)
    Waldrop, Megan A.
    Connolly, Anne M.
    Mendell, Jerry R.
    EXPERT OPINION ON ORPHAN DRUGS, 2021, 9 (7-10): : 199 - 204
  • [5] Onasemnogene Abeparvovec in Presymptomatic Spinal Muscular Atrophy (SMA): SPRINT Study Update in Children with Three Copies of SMN2
    Strauss, Kevin
    Muntoni, Francesco
    Farrar, Michelle
    Saito, Kayoko
    Mendell, Jerry
    Servais, Laurent
    McMillan, Hugh
    Finkel, Richard
    Swoboda, Kathryn
    Kwon, Jennifer
    Zaidman, Craig
    Chiriboga, Claudia
    Lannaccone, Susan
    Krueger, Jena
    Parsons, Julie
    Shieh, Perry
    Kavanagh, Sarah
    Chand, Deepa
    Tauscher-Wisniewski, Sitra
    McGill, Bryan
    Macek, Thomas
    NEUROLOGY, 2022, 98 (18)
  • [6] Outcomes in patients with spinal muscular atrophy and four or more SMN2 copies treated with onasemnogene abeparvovec: Findings from RESTORE
    Finkel, R.
    Benguerba, K.
    Reid, A.
    Raju, D.
    Faulkner, E.
    LaMarca, N.
    Servais, L.
    NEUROMUSCULAR DISORDERS, 2022, 32 : S59 - S59
  • [7] Outcomes for patients in the RESTORE registry with spinal muscular atrophy and four or more SMN2 gene copies treated with onasemnogene abeparvovec
    Tizzano, Eduardo F.
    Quijano-Roy, Susana
    Servais, Laurent
    Parsons, A.
    Parsons, Julie A.
    Aharoni, Sharon
    Lakhotia, Arpita
    Finkel, Richard S.
    EUROPEAN JOURNAL OF PAEDIATRIC NEUROLOGY, 2024, 53 : 18 - 24
  • [8] Bulbar Function in Children with Two or Three SMN2 Copies Who Received Onasemnogene Abeparvovec Presymptomatically for Spinal Muscular Atrophy
    McGrattan, Katlyn
    Shell, Richard
    Hurst-Davis, Rebecca
    Young, Sally Dunaway
    Baranello, Giovanni
    Lavrov, Arseniy
    O'Brien, Eamonn
    Wallach, Shiri
    LaMarca, Nicole
    Reyna, Sandra P.
    Darras, Basil
    NEUROLOGY, 2023, 100 (17)
  • [9] Outcomes in Patients with Spinal Muscular Atrophy and Four or More SMN2 Copies Treated with Onasemnogene Abeparvovec: Findings from RESTORE
    Finkel, Richard
    Benguerba, Kamal
    Gehani, Manish
    Raju, Dheeraj
    Faulkner, Eric
    LaMarca, Nicole
    Servais, Laurent
    NEUROLOGY, 2023, 100 (17)
  • [10] Outcomes in patients with spinal muscular atrophy (SMA) and four or more SMN2 copies treated with onasemnogene abeparvovec: findings from RESTORE
    Finkel, R.
    Benguerba, K.
    Gehani, M.
    Raju, D.
    Faulkner, E.
    LaMarca, N.
    Servais, L.
    NEUROMUSCULAR DISORDERS, 2023, 33 : S133 - S133