Muscle microRNAs in the cerebrospinal fluid predict clinical response to nusinersen therapy in type II and type III spinal muscular atrophy patients

被引:6
|
作者
Magen, I. [1 ]
Aharoni, S. [2 ]
Yacovzada, N. [1 ]
Latzer, I. Tokatly [3 ]
Alves, C. [4 ]
Sagi, L. [3 ]
Fattal-Valevski, A. [3 ]
Swoboda, K. [4 ]
Katz, J. [2 ]
Bruckheimer, E. [2 ]
Nevo, Y. [2 ]
Hornstein, E. [1 ]
机构
[1] Weizmann Inst Sci, Rehovot, Israel
[2] Schneider Childrens Med Ctr Israel, Petah Tiqwa, Israel
[3] Dana Dwek Childrens Hosp, Tel Aviv, Israel
[4] Massachusetts Gen Hosp, Boston, MA 02114 USA
关键词
D O I
10.1016/j.nmd.2022.07.089
中图分类号
R74 [神经病学与精神病学];
学科分类号
摘要
P.53
引用
收藏
页码:S62 / S62
页数:1
相关论文
共 50 条
  • [1] Muscle microRNAs in the cerebrospinal fluid predict clinical response to nusinersen therapy in type II and type III spinal muscular atrophy patients
    Magen, Iddo
    Aharoni, Sharon
    Yacovzada, Nancy Sarah
    Latzer, Itay Tokatly
    Alves, Christiano R. R.
    Sagi, Liora
    Fattal-Valevski, Aviva
    Swoboda, Kathryn J.
    Katz, Jacob
    Bruckheimer, Elchanan
    Nevo, Yoram
    Hornstein, Eran
    EUROPEAN JOURNAL OF NEUROLOGY, 2022, 29 (08) : 2420 - 2430
  • [2] Nusinersen Modulates Proteomics Profiles of Cerebrospinal Fluid in Spinal Muscular Atrophy Type 1 Patients
    Bianchi, Laura
    Sframeli, Maria
    Vantaggiato, Lorenza
    Vita, Gian Luca
    Ciranni, Annamaria
    Polito, Francesca
    Oteri, Rosaria
    Gitto, Eloisa
    Di Giuseppe, Fabrizio
    Angelucci, Stefania
    Versaci, Antonio
    Messina, Sonia
    Vita, Giuseppe
    Bini, Luca
    Aguennouz, M'hammed
    INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES, 2021, 22 (09)
  • [3] Nusinersen in pediatric and adult patients with type III spinal muscular atrophy
    Pera, Maria Carmela
    Coratti, Giorgia
    Bovis, Francesca
    Pane, Marika
    Pasternak, Amy
    Montes, Jacqueline
    Sansone, Valeria A.
    Dunaway Young, Sally
    Duong, Tina
    Messina, Sonia
    Mizzoni, Irene
    D'Amico, Adele
    Civitello, Matthew
    Glanzman, Allan M.
    Bruno, Claudio
    Salmin, Francesca
    Morando, Simone
    De Sanctis, Roberto
    Sframeli, Maria
    Antonaci, Laura
    Frongia, Anna Lia
    Rohwer, Annemarie
    Scoto, Mariacristina
    De Vivo, Darryl C.
    Darras, Basil T.
    Day, John
    Martens, William
    Patanella, Katia A.
    Bertini, Enrico
    Muntoni, Francesco
    Finkel, Richard
    Mercuri, Eugenio
    ANNALS OF CLINICAL AND TRANSLATIONAL NEUROLOGY, 2021, 8 (08): : 1622 - 1634
  • [4] Nusinersen treatment and cerebrospinal fluid neurofilaments: An explorative study on Spinal Muscular Atrophy type 3 patients
    Faravelli, Irene
    Meneri, Megi
    Saccomanno, Domenica
    Velardo, Daniele
    Abati, Elena
    Gagliardi, Delia
    Parente, Valeria
    Petrozzi, Lucia
    Ronchi, Dario
    Stocchetti, Nino
    Calderini, Edoardo
    D'Angelo, Grazia
    Chidini, Giovanna
    Prandi, Edi
    Ricci, Giulia
    Siciliano, Gabriele
    Bresolin, Nereo
    Comi, Giacomo Pietro
    Corti, Stefania
    Magri, Francesca
    Govoni, Alessandra
    JOURNAL OF CELLULAR AND MOLECULAR MEDICINE, 2020, 24 (05) : 3034 - 3039
  • [5] Clinical Outcomes in Patients with Spinal Muscular Atrophy Type 1 Treated with Nusinersen
    Mendonca, Rodrigo de Holanda
    Polido, Graziela Jorge
    Matsui, Ciro, Jr.
    Fontoura Solla, Davi Jorge
    Reed, Umbertina Conti
    Zanoteli, Edmar
    JOURNAL OF NEUROMUSCULAR DISEASES, 2021, 8 (02) : 217 - 224
  • [6] Cerebrospinal Fluid Proteomic Changes after Nusinersen in Patients with Spinal Muscular Atrophy
    Beaudin, Marie
    Kamali, Tahereh
    Tang, Whitney
    Hagerman, Katharine A.
    Dunaway Young, Sally
    Ghiglieri, Lisa
    Parker, Dana M.
    Lehallier, Benoit
    Tesi-Rocha, Carolina
    Sampson, Jacinda B.
    Duong, Tina
    Day, John W.
    JOURNAL OF CLINICAL MEDICINE, 2023, 12 (20)
  • [7] Cerebrospinal fluid characteristics of patients treated with intrathecal nusinersen for spinal muscular atrophy
    Orbach, Rotem
    Sagi, Liora
    Sadot, Efraim
    Latzer, Itay Tokatly
    Shtamler, Anna
    Zisberg, Tehila
    Fattal-Valevski, Aviva
    MUSCLE & NERVE, 2022, 66 (06) : 762 - 766
  • [8] Study on the efficacy, safety, and biomarkers of nusinersen in type II and III spinal muscular atrophy in children
    Chen, Liyuan
    Liu, Fen
    Fang, Danna
    Li, Jianwei
    FRONTIERS IN PEDIATRICS, 2023, 11
  • [9] Neurophysiological Characteristics in Type II and Type III 5q Spinal Muscular Atrophy Patients: Impact of Nusinersen Treatment
    Li, Dan
    Sun, Na
    Xiang, Li
    Liu, Jingjie
    Wang, Xueying
    Yang, Lin
    Huang, Shaoping
    DRUG DESIGN DEVELOPMENT AND THERAPY, 2024, 18 : 953 - 965
  • [10] Clinical and Electropthysiological outcomes of Spinal Muscular Atrophy type 1 patients treated with Nusinersen
    Kern-Smith, Emily
    Verma, Sumit
    NEUROLOGY, 2019, 92 (15)