FIREFISH Part 2: Efficacy and safety of risdiplam (RG7916) in infants with Type 1 spinal muscular atrophy (SMA)

被引：0

作者：

Baranello, Giovanni ^{[1
,2
]}

Servais, Laurent ^{[3
,4
]}

Masson, Riccardo ^{[1
]}

Mazurkiewicz-Beldzinska, Maria ^{[5
]}

Rose, Kristy ^{[6
]}

Vlodavets, Dmitry ^{[7
]}

Xiong, Hui ^{[8
]}

Zanoteli, Edmar ^{[9
]}

El-Khairi, Muna ^{[10
]}

Fuerst-Recktenwald, Sabine ^{[11
]}

Gerber, Marianne ^{[12
]}

Gorni, Ksenija ^{[13
]}

Kletzl, Heidemarie ^{[14
]}

Scalco, Renata ^{[11
]}

Darras, Basil T. ^{[15
]}

机构：

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

[2] UCL Great Ormond St Inst Child Hlth, Dubowitz Neuromuscular Ctr, London, England

[3] Ctr Hosp Reg Citadelle, Reference Ctr Neuromuscular Dis, Liege, Belgium

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

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

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

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

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

[9] Univ Sao Paulo, Hosp Clin, Dept Neurol, Sao Paulo, Brazil

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

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

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

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

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

[15] Harvard Med Sch, Boston Childrens Hosp, Boston, MA 02115 USA

来源：

关键词：

Infants; Neuromuscular diseases;

D O I：

10.1183/13993003.congress-2020.1172

中图分类号：

R56 [呼吸系及胸部疾病];

学科分类号：

摘要：

1172

引用

页数：3