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

共 50 条

[1] FIREFISH Parts 1 and 2: 24-Month Safety and Efficacy of Risdiplam in Type 1 SMA
Masson, R.
Boespflug-Tanguy, O.
Darras, B. T.
Day, J. W.
Deconinck, N.
Klein, A.
Mazurkiewicz-Bedziska, M.
Mercuri, E.
Rose, K.
Servais, L.
Vlodavets, D.
Xiong, H.
Zanoteli, E.
Dodman, A.
El-Khairi, M.
Gaki, E.
Gerber, M.
Gorni, K.
Kletzl, H.
Baranello, G.
EUROPEAN JOURNAL OF NEUROLOGY, 2021, 28 : 395 - 396
[2] FIREFISH Part 2: 24-month Efficacy and Safety of Risdiplam in Infants with Type 1 Spinal Muscular Atrophy (SMA)
Darras, Basil T.
Masson, Ricardo z
Mazurkiewicz-Beldzinska, Maria
Rose, Kristy
Xiong, Hui
Zanoteli, Edmar
Baranello, Giovanni
Vlodavets, Dmitry
Dodman, Angela
El-Khairi, Muna
Gerber, Marianne
Gorni, Ksenija
Kletzl, Heidemarie
Scalco, Renata S.
Servais, Laurent
NEUROLOGY, 2021, 96 (15)
[3] IREFISH Parts 1 and 2: 24-month safety and efficacy of risdiplam in type 1 spinal muscular atrophy (SMA)
Masson, R.
Boespflug-Tanguy, O.
Darras, B.
Day, J.
Deconinck, N.
Klein, A.
Mazurkiewicz-Beldzinska, M.
Mercuri, E.
Rose, K.
Servais, L.
Vlodavets, D.
Xiong, H.
Zanoteli, E.
Dodman, A.
El-Khairi, M.
Gaki, E.
Gerber, M.
Gorni, K.
Kletzl, H.
Baranello, G.
NEUROMUSCULAR DISORDERS, 2021, 31 : S134 - S134
[4] FIREFISH Parts 1 and 2: 36-month safety and efficacy of risdiplam in Type 1 spinal muscular atrophy (SMA)
Servais, L.
Baranello, G.
Boespflug-Tanguy, O.
Day, J.
Deconinck, N.
Klein, A.
Masson, R.
Mazurkiewicz-Beldzinska, M.
Mercuri, E.
Rose, K.
Vlodavets, D.
Xiong, H.
Zanoteli, E.
El-Khairi, M.
Gerber, M.
Gorni, K.
Kletzl, H.
Palfreeman, L.
Dodman, A.
Gaki, E.
Darras, B.
NEUROMUSCULAR DISORDERS, 2022, 32 : S88 - S88
[5] FIREFISH Parts 1 and 2: 36-month safety and efficacy of risdiplam in Type 1 spinal muscular atrophy (SMA)
Darras, Basil
Baranello, Giovanni
Boespflug-Tanguy, Odile
Day, John
Deconinck, Nicholas
Klein, Andrea
Masson, Ricardo
Mazurkiewicz-Beldzinska, Maria
Mercuri, Eugenio
Rose, Kristy
Vlodavets, Dmitry
Xiong, Hui
Zanoteli, Edmar
El-Khairi, Muna
Gerber, Marianne
Gorni, Ksenija
Kletzl, Heidemarie
Palfreeman, Laura
Dodman, Angela
Servais, Laurent
NEUROLOGY, 2023, 100 (17)
[6] FIREFISH Parts 1 and 2: 36-month safety and efficacy of risdiplam in Type 1 spinal muscular atrophy
Deconinck, N.
Baranello, G.
Boespflug-Tanguy, O.
Day, J.
Klein, A.
Masson, R.
Mazurkiewicz-Beldzinska, M.
Mercuri, E.
Rose, K.
Servais, L.
Vlodavets, D.
Xiong, H.
Zanoteli, E.
El-Khairi, M.
Gerber, M.
Gorni, K.
Kletzl, H.
Dodman, A.
Gaki, E.
Darras, B.
EUROPEAN JOURNAL OF NEUROLOGY, 2022, 29 : 279 - 279
[7] 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
[8] FIREFISH Parts 1 and 2: 4-year efficacy and safety of risdiplam in Type 1 spinal muscular atrophy (SMA)
Mazurkiewicz-Beldzinska, M.
Baranello, G.
Boespflug-Tanguy, O.
Day, J.
Deconinck, N.
Klein, A.
Masson, R.
Mercuri, E.
Rose, K.
Servais, L.
Vlodavets, D.
Xiong, H.
Zanoteli, E.
El-Khairi, M.
Gerber, M.
Gorni, K.
Kletzl, H.
Palfreeman, L.
Dodman, A.
Darras, B.
EUROPEAN JOURNAL OF NEUROLOGY, 2023, 30 : 196 - 196
[9] 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
[10] SUNFISH Part 2: 24-month efficacy and safety of risdiplam in patients with Type 2 or non-ambulant Type 3 SMA
Nascimento, A.
Day, J. W.
Deconinck, N.
Mazzone, E.
Oskoui, M.
Saito, K.
Vuillerot, C.
Baranello, G.
Boespflug-Tanguy, O.
Goemans, N.
Kirschner, J.
Kostera-Pruszczyk, A.
Servais, L.
Gerber, M.
Gorni, K.
Kletzl, H.
Martin, C.
Scalco, R. S.
Staunton, H.
Yeung, W. Yin
Mercuri, E.
EUROPEAN JOURNAL OF NEUROLOGY, 2021, 28 : 187 - 188

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