Gene Therapy (Onasemnogene Abeparvovec) Is Safe and Effective in Children with Spinal Muscular Atrophy Type 1 on Invasive Long-Term Ventilation

被引:0
|
作者
Narayan, O. [1 ]
Alajjuri, M. [2 ]
Abusamra, R. [3 ]
Mundada, V [4 ]
机构
[1] Amer Hosp Dubai, Pediat Pulmonol, Dubai, U Arab Emirates
[2] Univ Sharjah, Sharjah, U Arab Emirates
[3] Mediclin Hosp Dubai, Pediat Pulmonol, Dubai, U Arab Emirates
[4] Medcare Hosp Dubai, Pediat Neurol, Dubai, U Arab Emirates
关键词
D O I
暂无
中图分类号
R72 [儿科学];
学科分类号
100202 ;
摘要
H 139
引用
收藏
页码:S119 / S120
页数:2
相关论文
共 50 条
  • [41] Early Development of Spinal Deformities in Children Severely Affected with Spinal Muscular Atrophy after Gene Therapy with Onasemnogene Abeparvovec-Preliminary Results
    Soini, Venla
    Schreiber, Gudrun
    Wilken, Bernd
    Hell, Anna Kathrin
    CHILDREN-BASEL, 2023, 10 (06):
  • [42] Gene-replacement therapy (GRT) in spinal muscular atrophy type 1 (SMA1): long-term follow-up from the onasemnogene abeparvovec phase 1/2a clinical trial
    Mendell, J.
    Shell, R.
    Lehman, K.
    McColly, M.
    Lowes, L.
    Alfano, L.
    Miller, N.
    Iammarino, M.
    Church, K.
    Ogrinc, F.
    Ouyang, H.
    Kernbauer, E.
    Shah, S.
    L'Italien, J.
    Sproule, D.
    Feltner, D.
    Al-Zaidy, S.
    NEUROMUSCULAR DISORDERS, 2019, 29 : S184 - S184
  • [43] Survey of Physician Recommendation of Long-term Invasive Ventilation for Children with Spinal Muscular Atrophy Type I: Pilot survey
    Benson, R. C.
    Hsia, D. W.
    Hardy, K. A.
    AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE, 2010, 181
  • [44] Effects of long-term non-invasive ventilation on sleep structure in children with Spinal Muscular Atrophy type 2
    Verrillo, Elisabetta
    Pavone, Martino
    Bruni, Oliviero
    Ferri, Raffaele
    Caggiano, Serena
    Testa, Maria Beatrice Chiarini
    Cherchi, Claudio
    Cutrera, Renato
    SLEEP MEDICINE, 2019, 58 : 82 - 87
  • [45] Gene-replacement therapy (GRT) in spinal muscular atrophy type 1 (SMA1): Long-term follow-up from the onasemnogene abeparvovec phase 1/2A clinical trial
    Mendell, J. R.
    Shell, R.
    Lehman, K. J.
    McColly, M.
    Lowes, L. P.
    Alfano, L. N.
    Miller, N. F.
    Iammarino, M. A.
    Church, K.
    Spector, S. A.
    Ogrinc, F. G.
    Ouyang, H.
    Kernbauer, E.
    Shah, S.
    L'Italien, J.
    Sproule, D. M.
    Feltner, D. E.
    Al-Zaidy, S.
    JOURNAL OF THE NEUROLOGICAL SCIENCES, 2019, 405
  • [46] Combination therapy with onasemnogene and risdiplam in spinal muscular atrophy type 1
    Oechsel, Kelly F.
    Cartwright, Michael S.
    MUSCLE & NERVE, 2021, 64 (04) : 487 - 490
  • [47] Onasemnogene abeparvovec gene replacement therapy for the treatment of spinal muscular atrophy: a real-world observational study
    Ilaria Bitetti
    Valentina Lanzara
    Giovanna Margiotta
    Antonio Varone
    Gene Therapy, 2023, 30 : 592 - 597
  • [48] Gene-Replacement Therapy (GRT) in Spinal Muscular Atrophy Type 1 (SMA1): Long-Term Follow-Up From the Onasemnogene Abeparvovec Phase 1/2a Clinical Trial
    Mendell, J.
    Shell, R.
    Lehman, K.
    McColly, M.
    Lowes, L.
    Alfano, L.
    Miller, N.
    Iammarino, M.
    Church, K.
    Ogrinc, F.
    Ouyang, H.
    Kernbauer, E.
    Shah, S.
    L'Italien, J.
    Sproule, D.
    Feltner, D.
    Al-Zaidy, S.
    ANNALS OF NEUROLOGY, 2019, 86 : S126 - S126
  • [49] Onasemnogene Abeparvovec Gene-Replacement Therapy (GRT) in Pre-symptomatic Spinal Muscular Atrophy (SMA)
    Schultz, M.
    Swoboda, K.
    Farrar, M.
    McMillan, H.
    Parsons, J.
    Kernbauer, E.
    Farrow, M.
    Ogrinc, F.
    Kavanagh, S.
    Feltner, D.
    McGill, B.
    Spector, S.
    L'Italien, J.
    Sproule, D.
    Strauss, K.
    ANNALS OF NEUROLOGY, 2019, 86 : S133 - S133
  • [50] Onasemnogene Abeparvovec Gene Therapy and Risdiplam for the Treatment of Spinal Muscular Atrophy in Thailand: A Cost-Utility Analysis
    Khuntha, Sarayuth
    Prawjaeng, Juthamas
    Ponragdee, Kunnatee
    Sanmaneechai, Oranee
    Srinonprasert, Varalak
    Leelahavarong, Pattara
    APPLIED HEALTH ECONOMICS AND HEALTH POLICY, 2025, 23 (02) : 277 - 290