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：

EUROPEAN RESPIRATORY JOURNAL | 2020年 / 56卷

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

共 50 条

[1] FIREFISH Part 2: Efficacy and Safety of Risdiplam (RG7916) in Infants with Type 1 Spinal Muscular Atrophy (SMA)
Servais, Laurent
Baranello, Giovanni
Masson, Riccardo
Mazurkiewicz-Beldzinska, Maria
Rose, Kristy
Vlodavets, Dmitry
Xiong, Hui
Zanoteli, Edmar
El-Khairi, Muna
Fuerst-Recktenwald, Sabine
Gerber, Marianne
Gorni, Ksenija
Kletzl, Heidemarie
Scalco, Renata
Darras, Basil T.
NEUROLOGY, 2020, 94 (15)
[2] FIREFISH Part 2: Efficacy and safety of risdiplam (RG7916) in infants with Type 1 spinal muscular atrophy (SMA)
Servais, L.
Baranello, G.
Masson, R.
Mazurkiewicz-Beldzinska, M.
Rose, K.
Vlodavets, D.
Xiong, H.
Zanoteli, E.
El-Khairi, M.
Fuerst-Recktenwald, S.
Gerber, M.
Gorni, K.
Kletzl, H.
Scalco, R.
Darras, B. T.
EUROPEAN JOURNAL OF NEUROLOGY, 2020, 27 : 166 - 167
[3] FIREFISH Part 1: 24-month safety and exploratory outcomes of risdiplam (RG7916) in infants with Type 1 spinal muscular atrophy (SMA)
Baranello, G.
Bloespflug-Tanguy, O.
Darras, B.
Day, J.
Deconinck, N.
Klein, A.
Masson, R.
Mercuri, E.
Dodman, A.
El-Khairi, M.
Gerber, M.
Gorni, K.
Kletzl, H.
Scalco, R.
Servais, L.
NEUROMUSCULAR DISORDERS, 2020, 30 : S122 - S122
[4] FIREFISH Part 1: survival, ventilation and swallowing ability in infants with type 1 spinal muscular atrophy (SMA) treated with risdiplam (RG7916)
Vlodavets, D.
Servais, L.
Baranello, G.
Day, J. W.
Deconinck, N.
Mercuri, E.
Klein, A.
Darras, B.
Masson, R.
Kletzl, H.
Cleary, Y.
El-Khairi, M.
Seabrook, T.
Czech, C.
Gerber, M.
Nguyen, C.
Gelblin, K.
Gorni, K.
Khwaja, O.
EUROPEAN JOURNAL OF NEUROLOGY, 2019, 26 : 310 - 311
[5] FIREFISH Parts 1 and 2: 12-month pooled safety and efficacy outcomes of risdiplam (RG7916) in infants with Type 1 spinal muscular atrophy (SMA)
Servais, L.
Bloespflug-Tanguy, O.
Darras, B.
Day, J.
Deconinck, N.
Klein, A.
Masson, R.
Mazurkiewicz-Beldzinska, M.
Mercuri, E.
Rose, K.
Vlodavets, D.
Xiong, H.
Zanoteli, E.
Dodman, A.
El-Khairi, M.
Gerber, M.
Gorni, K.
Kletzl, H.
Scalco, R.
Baranello, G.
NEUROMUSCULAR DISORDERS, 2020, 30 : S126 - S126
[6] FIREFISH Part 1: 16-month safety and exploratory outcomes of risdiplam (RG7916) treatment in infants with type 1 spinal muscular atrophy
Baranello, G.
Servais, L.
Day, J.
Deconinck, N.
Mercuri, E.
Klein, A.
Darras, B.
Masson, R.
Kletzl, H.
Cleary, Y.
El-Khairi, M.
Seabrook, T.
Czech, C.
Gerber, M.
Nguyen, C.
Gelblin, K.
Gorni, K.
NEUROMUSCULAR DISORDERS, 2019, 29 : S184 - S184
[7] RAINBOWFISH: A study of risdiplam (RG7916) in infants with presymptomatic spinal muscular atrophy (SMA)
Servais, L.
Bertini, E.
Al-Muhaizea, M.
Nelson, L.
Prufer, A.
Vlodavets, D.
Wang, Y.
Zanoteli, E.
Burke, L.
El-Khairi, M.
Gorni, K.
Kletzl, H.
Gerber, M.
Scalco, R.
Finkel, R.
NEUROMUSCULAR DISORDERS, 2020, 30 : S127 - S127
[8] FIREFISH Part 1: Survival, Ventilation and Swallowing Ability in Infants with Type 1 SMA Receiving Risdiplam (RG7916)
Servais, Laurent
Baranello, Giovanni
Day, John W.
Deconinck, Nicolas
Mercuri, Eugenio
Klein, Andrea
Darras, Basil
Masson, Riccardo
Kletzl, Heidemarie
Cleary, Yumi
El-Khairi, Muna
Seabrook, Timothy
Czech, Christian
Gerber, Marianne
Nguyen, Coralie
Gelblin, Kristina
Gorni, Ksenija
Khwaja, Omar
NEUROLOGY, 2019, 92 (15)
[9] SUNFISH Part 2: Efficacy and safety of risdiplam (RG7916) in patients with Type 2 or non-ambulant Type 3 spinal muscular atrophy (SMA)
Mercuri, E.
Barisic, N.
Boespflug-Tanguy, O.
Day, J. W.
Deconinck, N.
Kostera-Pruszczyk, A.
Masson, R.
Mazzone, E.
Nascimento, A.
Saito, K.
Vlodavets, D.
Fuerst-Recktenwald, S.
Fuhrer, S.
Gerber, M.
Gorni, K.
Kletzl, H.
Martin, C.
Yeung, W. Y.
Vuillerot, C.
EUROPEAN JOURNAL OF NEUROLOGY, 2020, 27 : 869 - 869
[10] SUNFISH Part 2: Efficacy and Safety of Risdiplam (RG7916) in Patients with Type 2 or Non-Ambulant Type 3 Spinal Muscular Atrophy (SMA)
Mercuri, Eugenio
Barisic, Nina
Boespflug-Tanguy, Odile
Deconinck, Nicolas
Kostera-Pruszczyk, Anna
Masson, Riccardo
Mazzone, Elena
Nascimento, Andres
Saito, Kayoko
Vlodavets, Dmitry
Vuillerot, Carole
Fuerst-Recktenwald, Sabine
Fuhrer, Sibylle
Gerber, Marianne
Gorni, Ksenija
Kletzl, Heidemarie
Martin, Carmen
Yeung, Wai Yin
Day, John W.
NEUROLOGY, 2020, 94 (15)

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