Safety update: Risdiplam clinical trial program for spinal muscular atrophy (SMA)

被引：1

作者：

Baranello, G. ^{[1
,2
,3
]}

Chiriboga, C. ^{[4
]}

Servais, L. ^{[5
,6
,7
,8
]}

Darras, B. ^{[9
]}

Day, J. ^{[10
]}

Deconinck, N. ^{[11
,12
]}

Farrar, M. ^{[13
,14
]}

Finkel, R. ^{[15
]}

Bertini, E. ^{[16
]}

Kirschner, J. ^{[17
]}

Rasson, M. ^{[3
]}

Mazurkiewicz-Beldzinska, M. ^{[18
]}

Vlodavets, D. ^{[19
]}

Bader-Weder, S. ^{[20
]}

Gorni, K. ^{[21
]}

Jaber, B. ^{[20
]}

Yeung, W. ^{[22
]}

Papp, G. ^{[20
]}

Scalco, R. ^{[23
]}

Mercuri, E. ^{[24
,25
]}

机构：

[1] UCL, NIHR Gt Ormond St Hosp, Dubowitz Neuromusc Ctr, Biomed Res Ctr,Gt Ormond St Inst Child Hlth, London, England

[2] Gt Ormond Str Hosp Trust, London, England

[3] Fdn IRCCS Ist Neurol Carlo Besta, Dev Neurol Unit, Milan, Italy

[4] Columbia Univ, Dept Neurol, Irving Med Ctr, New York, NY USA

[5] Univ Oxford, Dept Paediat, MDUK Oxford Neuromusc Ctr, Oxford, England

[6] Univ Hosp Liege, Div Child Neurol, Dept Paediat, Ctr References Malad Neuromusculaires, Liege, Belgium

[7] Univ Liege, Liege, Belgium

[8] Hop Armand Trousseau, AP HP, I Mot, Inst Myol, Paris, France

[9] Harvard Med Sch, Boston Childrens Hosp, Dept Neurol, Boston, MA USA

[10] Stanford Univ, Dept Neurol, Palo Alto, CA USA

[11] Univ Libre Bruxelles, Queen Fabiola Childrens Univ Hosp, Ctr Reference Malad Neuromusculaires, Brussels, Belgium

[12] UZ Gent, Neuromusc Reference Ctr, Ghent, Belgium

[13] Sydney Childrens Hosp Network, Sydney, NSW, Australia

[14] UNSW Sydney, UNSW Med, Sydney, NSW, Australia

[15] St Jude Childrens Res Hosp, Ctr Expt Neurotherapeut, Memphis, TN USA

[16] Bambino Gesu Childrens Res Hosp IRCCS, Res Unit Neuromusc & Neurodegen Disorders, Rome, Italy

[17] Univ Freiburg, Fac Med, Med Ctr, Dept Neuropaediat & Muscle Disorders, Freiburg, Germany

[18] Med Univ Gdansk, Dept Develt Neurol, Gdansk, Poland

[19] Pirogov Russian Nat Res Med Univ, Russian Children Neuromusc Ctr, Veltischev Clin Paed Res Inst, Moscow, Russia

[20] F Hoffmann La Roche Ltd, Pharma Dev Safety, Basel, Switzerland

[21] F Hoffmann La Roche Ltd, PDMA Neurosci & Rare Dis, Basel, Switzerland

[22] Roche Prod Ltd, Welwyn Garden City, Herts, England

[23] F Hoffmann La Roche Ltd, Pharma Dev Neurol, Basel, Switzerland

[24] Catholic Univ, Paediat Neurol Inst, Rome, Italy

[25] Fdn Policlin Gemelli IRCCS, Nemo Pediat, Rome, Italy

来源：

NEUROMUSCULAR DISORDERS | 2023年 / 33卷

关键词：

D O I：

10.1016/j.nmd.2023.07.112

中图分类号：

R74 [神经病学与精神病学];

学科分类号：

摘要：

P230

引用

页码：S92 / S93

页数：2

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