Two-year efficacy and safety of risdiplam in patients with type 2 or non-ambulant type 3 spinal muscular atrophy (SMA) (vol 270, pg 2531, 2023)

被引：0

作者：

Oskoui, Maryam W. ^{[1
,2
]}

Day, John ^{[3
]}

Deconinck, Nicolas S. ^{[4
,5
,6
]}

Mazzone, Elena ^{[7
,8
]}

Nascimento, Andres ^{[9
]}

Saito, Kayoko ^{[10
]}

Vuillerot, Carole ^{[11
,12
]}

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

Goemans, Nathalie ^{[16
]}

Kirschner, Janbernd ^{[17
]}

Kostera-Pruszczyk, Anna ^{[18
]}

Servais, Laurent ^{[19
,20
,21
,22
]}

Papp, Gergely ^{[23
]}

Gorni, Ksenija ^{[24
]}

Kletzl, Heidemarie ^{[25
]}

Martin, Carmen ^{[26
]}

McIver, Tammy S. ^{[26
]}

Scalco, Renata ^{[27
]}

Staunton, Hannah ^{[26
]}

Yeung, Wai Yin ^{[26
]}

Fontoura, Paulo ^{[24
]}

Mercuri, Eugenio ^{[7
,8
]}

机构：

[1] McGill Univ, Dept Pediat, Montreal, PQ, Canada

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

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

[4] UZ Gent, Neuromuscular Reference Ctr, Ghent, Belgium

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

[6] Univ Libre Bruxelles, Queen Fabiola Childrens Univ Hosp, Serv Neurol Pediat, ULB, Brussels, Belgium

[7] Catholic Univ, Pediat Neurol Inst, Rome, Italy

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

[9] Hosp St Joan de Deu, Fdn St Joan de Deu, Neuropaediat Dept, Neuromuscular Unit,CIBERER ISC III, Barcelona, Spain

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

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

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

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

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

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

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

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

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

[19] Hop Armand Trousseau, I Mot, Paris, France

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

[21] Univ Hosp Liege, Ctr References Malad Neuromusculaires, Div Child Neurol, Liege, Belgium

[22] Univ Liege, Liege, Belgium

[23] F Hoffmann La Roche Ltd, Pharm Dev, Safety, Basel, Switzerland

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

[25] Roche Innovat Ctr Basel, Roche Pharmaceut Res & Early Dev, Basel, Switzerland

[26] Roche Prod Ltd, Welwyn Garden City, England

[27] F Hoffmann La Roche Ltd, Pharm Dev Neurol, Basel, Switzerland

来源：

JOURNAL OF NEUROLOGY | 2023年 / 270卷 / 5期

关键词：

D O I：

10.1007/s00415-023-11658-6

中图分类号：

R74 [神经病学与精神病学];

学科分类号：

摘要：

引用

页码：2547 / 2549

页数：3

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