Intravenous and intrathecal onasemnogene abeparvovec gene therapy in symptomatic and presymptomatic SMA: long-term follow-up study

被引：0
|
作者：
Darras, B. [1 ]
Farrar, M. [2 ,3 ]
Mercuri, E. [4 ,5 ]
Strauss, K. [6 ,7 ,8 ]
Day, J. [9 ]
Chien, Y. [10 ]
Masson, R. [11 ]
Bernardo, R. [12 ]
Alecu, I. [13 ]
Dodd, N. [14 ]
Mehl, L. [15 ]
Connolly, A. [16 ,17 ]
机构：
[1] Harvard Med Sch, Boston Childrens Hosp, Boston, MA USA
[2] Sydney Childrens Hosp Network, Sydney, NSW, Australia
[3] UNSW Sydney, Sch Clin Med, UNSW Med & Hlth, Sydney, NSW, Australia
[4] Catholic Univ, Dept Paediat Neurol, Rome, Italy
[5] Catholic Univ, Nemo Clin Ctr, Rome, Italy
[6] Clin Special Children, Strasburg, PA USA
[7] Penn Med Lancaster Gen Hosp, Lancaster, PA USA
[8] Univ Massachusetts, Sch Med, Worcester, MA USA
[9] Stanford Univ, Med Ctr, Stanford, CA USA
[10] Natl Taiwan Univ Hosp, Taipei, Taiwan
[11] Fdn IRCCS Ist Neurol Carlo Besta, Milan, Italy
[12] Novartis Global Drug Dev Neurosci & Gene Therap, E Hanover, NJ USA
[13] Novartis Pharmaceut, Basel, Switzerland
[14] Novartis Gene Therapies Inc, Bannockburn, IL USA
[15] Novartis, BioMed Res, Cambridge, MA USA
[16] Nationwide Childrens Hosp, Ctr Gene Therapy, Columbus, OH USA
[17] Ohio State Univ, Coll Med, Columbus, OH USA
来源：
NEUROMUSCULAR DISORDERS | 2024年 / 43卷
关键词：
D O I：
暂无
中图分类号：
R74 [神经病学与精神病学];
学科分类号：
摘要：
179P
引用
收藏
页数：1
相关论文
共 50 条
  • [31] Long-term follow-up of severely symptomatic women with adenomyoma treated with combination therapy
    Liu, Wei-Min
    Chen, Ching-Hui
    Chiu, Li-Hsuan
    Tzeng, Chii-Ruey
    TAIWANESE JOURNAL OF OBSTETRICS & GYNECOLOGY, 2013, 52 (01): : 85 - 89
  • [32] LONG-TERM ANTICOAGULANT THERAPY IN ANGINA PECTORIS - FOLLOW-UP STUDY
    BORCHGREVINK, CF
    LANCET, 1962, 1 (7227): : 449 - &
  • [33] ECLAMPSIA - A LONG-TERM FOLLOW-UP STUDY
    BRYANS, CI
    ZUSPAN, F
    SOUTHERLAND, WL
    OBSTETRICS AND GYNECOLOGY, 1963, 21 (06): : 701 - &
  • [34] LYMPHANGIOMA - LONG-TERM FOLLOW-UP STUDY
    SAIJO, M
    MUNRO, IR
    MANCER, K
    PLASTIC AND RECONSTRUCTIVE SURGERY, 1975, 56 (06) : 642 - 651
  • [35] Long-term follow-up of the STOPAGO study
    Cottu, Adrien
    Guillet, Stephanie
    Viallard, Jean-Francois
    Riviere, Etienne
    Cheze, Stephane
    Gobert, Delphine
    Neel, Antoine
    Graveleau, Julie
    Marolleau, Jean-Pierre
    Lefrere, Francois
    Moulis, Guillaume
    Lega, Jean-Christophe
    Moignet-Autrel, Aline
    Robbins, Ailsa
    Crickx, Etienne
    Boutin, Emmanuelle
    Noel, Nicolas
    Malphettes, Marion
    Galicier, Lionel
    Audia, Sylvain
    Bonnotte, Bernard
    Lambotte, Olivier
    Fain, Olivier
    Gerfaud-Valentin, Mathieu
    Terriou, Louis
    Martis, Nihal
    Morin, Anne-Sophie
    Perlat, Antoinette
    Le Gallou, Thomas
    Roy-Peaud, Frederique
    Puyade, Matthieu
    Comont, Thibault
    Limal, Nicolas
    Languille, Laetitia
    Michel, Marc
    Godeau, Bertrand
    Mahevas, Matthieu
    BLOOD, 2025, 145 (02) : 244 - 247
  • [36] PATIENT FOLLOW-UP IN A LONG-TERM STUDY
    DALLAS, J
    JOHNS HOPKINS MEDICAL JOURNAL, 1971, 128 (05): : 244 - &
  • [37] LONG-TERM FOLLOW-UP AND THERAPY IN MULTIPLE MYELOMA
    VELEZGAR.E
    MALDONADO, N
    CANCER, 1971, 27 (01) : 44 - +
  • [38] Rituximab therapy in pemphigus: A long-term follow-up
    Loi, Camilla
    Magnano, Michela
    Ravaioli, Giulia Maria
    Sacchelli, Lidia
    Patrizi, Annalisa
    Bardazzi, Federico
    DERMATOLOGIC THERAPY, 2019, 32 (01)
  • [39] Long-term follow-up of aggressive therapy in neurosarcoidosis
    Yandora, K
    Valeri, A
    Schramke, CJ
    Scott, TF
    NEUROLOGY, 2006, 66 (05) : A188 - A188
  • [40] Long-term follow-up of occlusion therapy in amblyopia
    Leiba, H
    Shimshoni, M
    Oliver, M
    Gottesman, N
    Levartovsky, S
    OPHTHALMOLOGY, 2001, 108 (09) : 1552 - 1555
12345