FIREFISH Parts 1 and 2: 24-month Safety and Efficacy of Risdiplam in Type 1 SMA

被引：0

作者：

Darras, Basil T. ^{[1
]}

Boespflug-Tanguy, Odile ^{[2
,3
]}

Day, John W. ^{[4
]}

Deconinck, Nicolas ^{[5
,6
]}

Klein, Andrea ^{[7
,8
]}

Masson, Riccardo ^{[9
]}

Mazurkiewicz-Beldzinska, Maria ^{[10
]}

Mercuri, Eugenio ^{[11
,12
]}

Rose, Kristy ^{[13
]}

Servais, Laurent ^{[2
,14
,15
,16
]}

Vlodavets, Dmitry ^{[17
]}

Xiong, Hui ^{[18
]}

Zanoteli, Edmar ^{[19
]}

Dodman, Angela ^{[20
]}

El-Khairi, Muna ^{[21
]}

Gaki, Eleni ^{[21
]}

Gerber, Marianne ^{[22
]}

Gorni, Ksenija ^{[23
]}

Kletzl, Heidemarie ^{[24
]}

Baranello, Giovanni ^{[9
,25
,26
]}

机构：

[1] Harvard Med Sch, Boston Childrens Hosp, Dept Neurol, Boston, MA 02115 USA

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

[3] Univ Paris, NeuroDiderot, UMR 1141, Paris, France

[4] Stanford Univ, Dept Neurol, Stanford, CA 94305 USA

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

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

[7] Univ Childrens Hosp Basel, Paediat Neurol, Basel, Switzerland

[8] Univ Bern, Inselspital, Paediat Neurol, Bern, Switzerland

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

[10] Med Univ Gdansk, Dept Dev Neurol, Gdansk, Poland

[11] Catholic Univ, Pediat Neurol Inst, Washington, DC USA

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

[13] Childrens Hosp Westmead, Paediat Gait Anal Serv New South Wales, Westmead, NSW, Australia

[14] MDUK Oxford Neuromuscular Ctr, Dept Paediat, Oxford, England

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

[16] Univ Liege, Liege, Belgium

[17] Pirogov Russian Natl Res Med Univ, Russian Children Neuromuscular Ctr, Veltischev Clin Pediat Res Inst, Moscow, Russia

[18] Peking Univ First Hosp, Dept Pediat, Beijing, Peoples R China

[19] Univ Sao Paulo, Fac Med, Dept Neurol, FMUSP, Sao Paulo, Brazil

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

[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

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

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

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

来源：

NEUROLOGY | 2022年 / 98卷 / 18期

关键词：

D O I：

暂无

中图分类号：

R74 [神经病学与精神病学];

学科分类号：

摘要：

S39.005

引用

页数：6

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