JEWELFISH: 24-month safety and pharmacodynamic data in non-treatment-naive patients with spinal muscular atrophy (SMA)

被引：0
|
作者：
Chiriboga, C. [1 ]
Bruno, C. [2 ]
Duong, T. [3 ]
Fischer, D. [4 ]
Kirschner, J. [5 ]
Scoto, M. [6 ]
Mercuri, E. [7 ]
Gerber, M. [8 ]
Gorni, K. [8 ]
Kletzl, H. [9 ]
Carruthers, I. [10 ]
Martin, C. [10 ]
Gidaro, T. [8 ]
Muntoni, F. [6 ]
机构：
[1] Columbia Irving Med Ctr, New York, NY USA
[2] Ist Giannina Gaslini, Genoa, Italy
[3] Stanford Univ, Palo Alto, CA 94304 USA
[4] Univ Childrens Hosp, Basel, Switzerland
[5] Univ Freiburg, Freiburg, Germany
[6] UCL, London, England
[7] Catholic Univ, Rome, Italy
[8] F Hoffmann La Roche Ltd, Basel, Switzerland
[9] Roche Innovat Ctr Basel, Basel, Switzerland
[10] Roche Prod Ltd, Welwyn Garden City, Herts, England
来源：
NEUROMUSCULAR DISORDERS | 2022年 / 32卷
关键词：
D O I：
10.1016/j.nmd.2022.07.195
中图分类号：
R74 [神经病学与精神病学];
学科分类号：
摘要：
P.110
引用
收藏
页码：S88 / S88
页数：1
相关论文
共 50 条
  • [1] JEWELFISH: 24-month Safety, Pharmacodynamic and Exploratory Efficacy Data in Non-Treatment-Naive Patients with Spinal Muscular Atrophy (SMA) Receiving Treatment with Risdiplam
    Chiriboga, Claudia
    Bruno, Claudio
    Duong, Tina
    Fischer, Dirk
    Kirschner, Janbernd
    Scoto, Mariacristina
    Mercuri, Eugenio
    Gerber, Marianne
    Gorni, Ksenija
    Kletzl, Heidemarie
    Carruthers, Imogen
    Martin, Carmen
    Gidaro, Teresa
    Muntoni, Francesco
    NEUROLOGY, 2023, 100 (17)
  • [2] 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)
  • [3] 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
  • [4] JEWELFISH: Safety and Pharmacodynamic Data in Non-Naive Patients with Spinal Muscular Atrophy (SMA) Receiving Treatment with Risdiplam (RG7916)
    Chiriboga, Claudia
    Bruno, Claudio
    Day, John W.
    Duong, Tina
    Fischer, Dirk
    Kirschner, Janbernd
    Muntoni, Francesco
    Fuerst-Recktenwald, Sabine
    Gerber, Marianne
    Gorni, Ksenija
    Kletzl, Heidemarie
    Warren, Francis
    Yeung, Wai Yin
    Mercuri, Eugenio
    NEUROLOGY, 2020, 94 (15)
  • [5] 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)
  • [6] Jewelfish: Safety and pharmacodynamic data in non-naive patients with spinal muscular atrophy receiving treatment with risdiplam (RG7916)
    Chiriboga, C.
    Bruno, C.
    Duong, T.
    Fischer, D.
    Kirschner, J.
    Mercuri, E.
    Gerber, M.
    Gorni, K.
    Kletzl, H.
    McIver, T.
    Scalco, R.
    Warren, F.
    Scoto, M.
    NEUROMUSCULAR DISORDERS, 2020, 30 : S126 - S127
  • [7] JEWELFISH: 12-month safety, pharmacodynamic and exploratory efficacy of risdiplam in non-naive patients with SMA
    Bruno, C.
    Chiriboga, C. A.
    Duong, T.
    Fischer, D.
    Kirschner, J.
    Mercuri, E.
    Gerber, M.
    Gorni, K.
    Kletzl, H.
    Carruthers, I.
    Scalco, R. S.
    Warren, F.
    Muntoni, F.
    EUROPEAN JOURNAL OF NEUROLOGY, 2021, 28 : 396 - 397
  • [8] JEWELFISH: SAFETY, PHARMACODYNAMIC AND EXPLORATORY EFFICACY DATA IN NON-NAIVE PATIENTS WITH SMA RECEIVING RISDIPLAM
    Scoto, Mariacristina
    Bruno, Claudio
    Fischer, Dirk
    Kirschner, Janbernd
    Mercuri, Eugenio
    Carruthers, Imogen
    Gerber, Marianne
    Kletzl, Heidemarie
    Warren, Francis
    Chiriboga, Claudia
    JOURNAL OF NEUROLOGY NEUROSURGERY AND PSYCHIATRY, 2022, 93 (09):
  • [9] 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
  • [10] IREFISH Parts 1 and 2: 24-month safety and efficacy of risdiplam in type 1 spinal muscular atrophy (SMA)
    Masson, R.
    Boespflug-Tanguy, O.
    Darras, B.
    Day, J.
    Deconinck, N.
    Klein, A.
    Mazurkiewicz-Beldzinska, M.
    Mercuri, E.
    Rose, K.
    Servais, L.
    Vlodavets, D.
    Xiong, H.
    Zanoteli, E.
    Dodman, A.
    El-Khairi, M.
    Gaki, E.
    Gerber, M.
    Gorni, K.
    Kletzl, H.
    Baranello, G.
    NEUROMUSCULAR DISORDERS, 2021, 31 : S134 - S134
12345