SUNFISH Parts 1 and 2: 4-year Efficacy and Safety Data of Risdiplam in Types 2 and 3 Spinal Muscular Atrophy (SMA)

被引：1

作者：

Servais, Laurent ^{[1
,2
,3
]}

Oskoui, Maryam ^{[4
]}

Day, John ^{[5
]}

Deconinck, Nicholas ^{[6
,7
]}

Mazzone, Elena ^{[8
]}

Nascimento, Andres ^{[9
]}

Saito, Kyoko ^{[10
]}

Vuillerot, Carole ^{[11
,12
]}

Baranello, Giovanni ^{[13
,14
,15
]}

Boespflug-Tanguy, Odile ^{[16
]}

Goemans, Nathalie ^{[17
]}

Kirschner, Janbernd ^{[18
]}

Kostera-Pruszczyk, Anna ^{[19
]}

Braid, Jessica ^{[20
]}

Papp, Gergely ^{[21
]}

Gorni, Ksenija ^{[22
]}

Martin, Carmen ^{[20
]}

Scalco, Renata ^{[23
]}

Yeung, Wai Yin ^{[20
]}

Mercuri, Eugenio ^{[21
]}

机构：

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

[2] Univ Oxford, Dept Paediat, MDUK Oxford Neuromuscular Ctr, Oxford, England

[3] Univ Liege, Univ Hosp Liege, Div Child Neurol, Ctr References Malad Neuromusculaires,Dept Pediat, Liege, Belgium

[4] McGill Univ, Dept Pediat & Neurol Neurosurg, Montreal, PQ, Canada

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

[6] ULB, Childrens Univ Hosp, Ctr Reference Malad Neuromusculaires, Brussels, Belgium

[7] UZ Gent, Neuromuscular Ctr, Ghent, Belgium

[8] Fondazione Policlinico Gemelli IRCCS, Cathol Univ & Nemo Pediat, Pediat Neurol Inst, Rome, Italy

[9] Fdn Sant Joan Deu, Hosp Sant Joan Deu, Neuromuscular Unit,Neuropaediat Dept, CIBERER,ISCIII, Barcelona, Spain

[10] Tokyo Womens Med Univ, Inst Med Genet, Tokyo, Japan

[11] CHU Lyon, Dept Pediat Phys Med & Rehabil, Hop Mere Enfant, Lyon, France

[12] Univ Lyon, CNRS UMR 5310 INSERM U1217, Neuromyogen Inst, Lyon, France

[13] UCL, NIHR Great Ormond St Hosp, Dubowitz Neuromuscular Ctr, Biomed Res Ctr,Great Ormond St Inst Child Hlth, London, England

[14] Great Ormond St Hosp Trust, London, England

[15] Fondazione IRCCS Ist Neurol Carlo Besta, Dev Neurol Unit, Milan, Italy

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

[17] Univ Paris Cite, UMR 1141, Neurodiderot, Paris, France

[18] Univ Hosp Leuven, Dept Paediat & Child Neurol, Neuromuscular Reference Ctr, Leuven, Belgium

[19] Univ Freiburg, Dept Neuropediat & Muscle Disorders, Med Ctr, Freiburg, Germany

[20] Med Univ Warsaw, Dept Neurol, Warsaw, Poland

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

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

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

来源：

NEUROLOGY | 2023年 / 100卷 / 17期

关键词：

D O I：

10.1212/WNL.0000000000203570

中图分类号：

R74 [神经病学与精神病学];

学科分类号：

摘要：

S34.009

引用

页数：7

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