More is needed to complement the available therapies of spinal muscular atrophy

被引：13
|
作者：
Singh, Ravindra N. [1 ]
机构：
[1] Iowa State Univ, Dept Biomed Sci, Ames, IA 50011 USA
来源：
FUTURE MEDICINAL CHEMISTRY | 2019年 / 11卷 / 22期
关键词：
AAV9; gene therapy; ISS-N1; nusinersen; oligonucleotide; spinal muscular atrophy (SMA); Spinraza; survival motor neuron (SMN); SURVIVAL MOTOR-NEURON; DISCOVERY; MODIFIER; DISEASE;
D O I：
10.4155/fmc-2019-0239
中图分类号：
R914 [药物化学];
学科分类号：
100701 ;
摘要：
[No abstract available]
引用
收藏
页码：2873 / 2876
页数：4
相关论文
共 50 条
  • [21] The End of the Beginning: The Journey to Molecular Therapies for Spinal Muscular Atrophy
    Pacak, Christina A.
    Kang, Peter B.
    PEDIATRIC NEUROLOGY, 2020, 102 : 1 - 2
  • [22] Development and preclinical evaluation of therapies for spinal muscular atrophy in mice
    Burghes, Arthur
    Foust, Kevin
    McGovern, Vicki
    Poresnsky, Paul
    Bevan, Adam
    Duque, Sandra
    Le, Thanh
    Iyer, Chitra
    Laporte, Aurelie
    Alwine, Isaac
    Mitrpant, Chalermchai
    Wilton, Steve
    Kaspar, Brian
    TRANSGENIC RESEARCH, 2011, 20 (05) : 1143 - 1144
  • [23] New treatments in spinal muscular atrophy: an overview of currently available data
    Ramdas, Sithara
    Servais, Laurent
    EXPERT OPINION ON PHARMACOTHERAPY, 2020, 21 (03) : 307 - 315
  • [24] Mortality in Spinal Muscular Atrophy in the era of disease-modifying therapies
    Finnegan, R.
    Rohwer, A.
    Scoto, M.
    Main, M.
    Baranello, G.
    Manzur, A.
    Muntoni, F.
    Munot, P.
    NEUROMUSCULAR DISORDERS, 2024, 43
  • [25] RNA splicing: More clues from spinal muscular atrophy
    Matera, AG
    CURRENT BIOLOGY, 1999, 9 (04) : R140 - R142
  • [26] Combination molecular therapies for spinal muscular atrophy: How much is enough?
    Mathews, Katherine D.
    Iannaccone, Susan T.
    MUSCLE & NERVE, 2021, 63 (03) : 279 - 281
  • [27] Spinal Muscular Atrophy: More than a Disease of Motor Neurons?
    Nash, L. A.
    Burns, J. K.
    Chardon, J. Warman
    Kothary, R.
    Parks, R. J.
    CURRENT MOLECULAR MEDICINE, 2016, 16 (09) : 779 - 792
  • [28] Novel therapies for spinal muscular atrophy are likely changing the patient phenotype
    Tu-Lan Vu-Han
    Wei, Claudia
    Pumberger, Matthias
    SPINE JOURNAL, 2020, 20 (12): : 1893 - 1898
  • [29] Onasemnogene abeparvovec in spinal muscular atrophy : predictors of efficacy and safety in naive patients with spinal muscular atrophy and following switch from other therapies
    Pane, Marika
    Berti, Beatrice
    Capasso, Anna
    Coratti, Giorgia
    Varone, Antonio
    D'Amico, Adele
    Messina, Sonia
    Masson, Riccardo
    Sansone, Valeria Ada
    Donati, Maria Alice
    Agosto, Caterina
    Bruno, Claudio
    Ricci, Federica
    Pini, Antonella
    Gagliardi, Delio
    Filosto, Massimiliano
    Corti, Stefania
    Leone, Daniela
    Palermo, Concetta
    Onesimo, Roberta
    De Sanctis, Roberto
    Ricci, Martina
    Bitetti, Ilaria
    Sframeli, Maria
    Dosi, Claudia
    Albamonte, Emilio
    Ticci, Chiara
    Brolatti, Noemi
    Bertini, Enrico
    Finkel, Richard
    Mercuri, Eugenio
    ECLINICALMEDICINE, 2023, 59
  • [30] Spinal muscular atrophy
    Chokri, M.
    Salma, S.
    JOURNAL OF THE NEUROLOGICAL SCIENCES, 2019, 405
12345