Risdiplam in Patients Previously Treated with Other Therapies for Spinal Muscular Atrophy: An Interim Analysis from the JEWELFISH Study

被引：0
|
作者：
Claudia A. Chiriboga
Claudio Bruno
Tina Duong
Dirk Fischer
Eugenio Mercuri
Janbernd Kirschner
Anna Kostera-Pruszczyk
Birgit Jaber
Ksenija Gorni
Heidemarie Kletzl
Imogen Carruthers
Carmen Martin
Francis Warren
Renata S. Scalco
Kathryn R. Wagner
Francesco Muntoni
机构：
[1] Columbia University Irving Medical Center,Department of Neurology
[2] University of Genoa,Centre of Translational and Experimental Myology, IRCCS Istituto Giannina Gaslini, and Department of Neuroscience, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health
[3] Stanford University,DINOGMI
[4] University Children’s Hospital Basel,Department of Neurology
[5] University of Basel,Division of Neuropediatrics
[6] Fondazione Policlinico Gemelli IRCCS,Pediatric Neurology Institute, Catholic University and Nemo Pediatrico
[7] Medical Center-University of Freiburg,Department of Neuropediatrics and Muscle Disorders, Faculty of Medicine
[8] Medical University of Warsaw,Department of Neurology
[9] ERN EURO-NMD,Roche Pharmaceutical Research and Early Development
[10] Pharma Development,Product Development Neuroscience
[11] Safety,The Dubowitz Neuromuscular Centre, NIHR Great Ormond Street Hospital Biomedical Research Centre
[12] F. Hoffmann-La Roche Ltd,undefined
[13] PDMA Neuroscience and Rare Disease,undefined
[14] F. Hoffmann-La Roche Ltd,undefined
[15] Roche Innovation Center Basel,undefined
[16] Roche Products Ltd,undefined
[17] F. Hoffmann-La Roche Ltd,undefined
[18] Great Ormond Street Institute of Child Health University College London,undefined
[19] and Great Ormond Street Hospital Trust,undefined
来源：
Neurology and Therapy | 2023年 / 12卷
关键词：
Evrysdi; Pharmacodynamics; Risdiplam; Safety; Spinal muscular atrophy;
D O I：
暂无
中图分类号：
学科分类号：
摘要：
引用
收藏
页码：543 / 557
页数：14
相关论文
共 50 条
  • [1] Risdiplam in Patients Previously Treated with Other Therapies for Spinal Muscular Atrophy: An Interim Analysis from the JEWELFISH Study
    Chiriboga, Claudia A. A.
    Bruno, Claudio
    Duong, Tina
    Fischer, Dirk
    Mercuri, Eugenio
    Kirschner, Janbernd
    Kostera-Pruszczyk, Anna
    Jaber, Birgit
    Gorni, Ksenija
    Kletzl, Heidemarie
    Carruthers, Imogen
    Martin, Carmen
    Warren, Francis
    Scalco, Renata S. S.
    Wagner, Kathryn R. R.
    Muntoni, Francesco
    NEUROLOGY AND THERAPY, 2023, 12 (02) : 543 - 557
  • [2] Correction to: Risdiplam in Patients Previously Treated with Other Therapies for Spinal Muscular Atrophy: An Interim Analysis from the JEWELFISH Study
    Claudia A. Chiriboga
    Claudio Bruno
    Tina Duong
    Dirk Fischer
    Eugenio Mercuri
    Janbernd Kirschner
    Anna Kostera-Pruszczyk
    Birgit Jaber
    Ksenija Gorni
    Heidemarie Kletzl
    Imogen Carruthers
    Carmen Martin
    Francis Warren
    Renata S. Scalco
    Kathryn R. Wagner
    Francesco Muntoni
    Neurology and Therapy, 2023, 12 : 1799 - 1801
  • [3] Risdiplam in Patients Previously Treated with Other Therapies for Spinal Muscular Atrophy: An Interim Analysis from the JEWELFISH Study (vol 12, pg 543, 2023)
    Chiriboga, Claudia
    Bruno, Claudio
    Duong, Tina
    Fischer, Dirk
    Mercuri, Eugenio
    Kirschner, Janbernd
    Kostera-Pruszczyk, Anna
    Jaber, Birgit A.
    Gorni, Ksenija
    Kletzl, Heidemarie
    Carruthers, Imogen
    Martin, Carmen
    Warren, Francis
    Scalco, Renata
    Wagner, Kathryn
    Muntoni, Francesco
    JEWELFISH Study Grp
    NEUROLOGY AND THERAPY, 2023, 12 (05) : 1799 - 1801
  • [4] JEWELFISH: safety and pharmacodynamic data in patients with spinal muscular atrophy (SMA) receiving treatment with risdiplam (RG7916) that have previously been treated with nusinersen
    Chiriboga, C.
    Mercuri, E.
    Fischer, D.
    Day, J.
    Kraus, D.
    Yeung, W.
    Kletzl, H.
    Gerber, M.
    Cleary, Y.
    Gorni, K.
    NEUROMUSCULAR DISORDERS, 2019, 29 : S187 - S187
  • [5] Children with spinal muscular atrophy treated with nusinersen/risdiplam
    Chacko, A.
    Sly, P.
    Deegan, S.
    Young, E.
    Gauld, L.
    RESPIROLOGY, 2023, 28 : 204 - 205
  • [6] Children With Spinal Muscular Atrophy Treated With Nusinersen/Risdiplam
    Chacko, A.
    Sly, P. D.
    Deegan, S.
    Young, E.
    Gauld, L.
    AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE, 2023, 207
  • [7] JEWELFISH: Safety and Pharmacodynamic Data in Non-Naive Patients with Spinal Muscular Atrophy (SMA) Receiving Treatment with Risdiplam
    Chiriboga, Claudia A.
    Bruno, Claudio
    Duong, Tina
    Fischer, Dirk
    Kirschner, Janbernd
    Mercuri, Eugenio
    Gerber, Marianne
    Gorni, Ksenija
    Kletzl, Heidemarie
    McIver, Tammy
    Scalco, Renata S.
    Warren, Francis
    Scoto, Mariacristina
    NEUROLOGY, 2021, 96 (15)
  • [8] Interim results from the RESPOND study evaluating nusinersen in children with spinal muscular atrophy previously treated with onasemnogene abeparvovec
    Parsons, J.
    Kuntz, N.
    Brandsema, J.
    Proud, C.
    Finkel, R.
    Swoboda, K.
    Masson, R.
    Foster, R.
    Liu, Y.
    Makepeace, C.
    Singhi, S.
    Paradis, A.
    Berger, Z.
    Rane, S.
    Somera-Molina, K.
    NEUROMUSCULAR DISORDERS, 2023, 33 : S87 - S87
  • [9] JEWELFISH: Safety, pharmacodynamic and exploratory efficacy data in non-naive patients with spinal muscular atrophy (SMA) receiving risdiplam
    Chiriboga, C.
    Bruno, C.
    Duong, T.
    Fischer, D.
    Kirschner, J.
    Mercuri, E.
    Gerber, M.
    Gorni, K.
    Kletzl, H.
    Carruthers, I.
    Martin, C.
    Warren, F.
    Scoto, M.
    NEUROMUSCULAR DISORDERS, 2021, 31 : S134 - S135
  • [10] JEWELFISH: Safety, Pharmacodynamic and Exploratory Efficacy Data in Non -Na ve Patients with Spinal Muscular Atrophy (SMA) Receiving Treatment with Risdiplam
    Chiriboga, Claudia A.
    Bruno, Claudio
    Duong, Tina
    Fischer, Dirk
    Kirschner, Janbernd
    Mercuri, Eugenio
    Gerber, Marianne
    Gorni, Ksenija
    Kletzl, Heidemarie
    Carruthers, Imogen
    Martin, Carmen
    Warren, Francis
    Scoto, Mariacristina
    NEUROLOGY, 2022, 98 (18)
12345