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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[1] Two-year efficacy and safety of risdiplam in patients with type 2 or non-ambulant type 3 spinal muscular atrophy (SMA)
Oskoui, Maryam
Day, John W.
Deconinck, Nicolas
Mazzone, Elena S.
Nascimento, Andres
Saito, Kayoko
Vuillerot, Carole
Baranello, Giovanni
Goemans, Nathalie
Kirschner, Janbernd
Kostera-Pruszczyk, Anna
Servais, Laurent
Papp, Gergely
Gorni, Ksenija
Kletzl, Heidemarie
Martin, Carmen
McIver, Tammy
Scalco, Renata S.
Staunton, Hannah
Yeung, Wai Yin
Fontoura, Paulo
Mercuri, Eugenio
SUNFISH Working Grp
JOURNAL OF NEUROLOGY, 2023, 270 (05) : 2531 - 2546
[2] Two-year efficacy and safety of risdiplam in patients with type 2 or non-ambulant type 3 spinal muscular atrophy (SMA)
Maryam Oskoui
John W. Day
Nicolas Deconinck
Elena S. Mazzone
Andres Nascimento
Kayoko Saito
Carole Vuillerot
Giovanni Baranello
Nathalie Goemans
Janbernd Kirschner
Anna Kostera-Pruszczyk
Laurent Servais
Gergely Papp
Ksenija Gorni
Heidemarie Kletzl
Carmen Martin
Tammy McIver
Renata S. Scalco
Hannah Staunton
Wai Yin Yeung
Paulo Fontoura
Eugenio Mercuri
Journal of Neurology, 2023, 270 : 2531 - 2546
[3] Correction to: Two‑year efficacy and safety of risdiplam in patients with type 2 or non‑ambulant type 3 spinal muscular atrophy (SMA)
Maryam Oskoui
John W. Day
Nicolas Deconinck
Elena S. Mazzone
Andres Nascimento
Kayoko Saito
Carole Vuillerot
Giovanni Baranello
Nathalie Goemans
Janbernd Kirschner
Anna Kostera-Pruszczyk
Laurent Servais
Gergely Papp
Ksenija Gorni
Heidemarie Kletzl
Carmen Martin
Tammy McIver
Renata S. Scalco
Hannah Staunton
Wai Yin Yeung
Paulo Fontoura
Eugenio Mercuri
Journal of Neurology, 2023, 270 : 2547 - 2549
[4] SUNFISH Part 2: 24-month Efficacy and Safety of Risdiplam in Patients with Type 2 or Non-ambulant Type 3 Spinal Muscular Atrophy (SMA)
Oskoui, Maryam
Day, John W.
Deconinck, Nicolas
Mazzone, Elena
Nascimento, Andres
Saito, Kayoko
Vuillerot, Carole
Baranello, Giovanni
Bloespflug-Tanguy, Odile
Goemans, Nathalie
Kirschner, Janbernd
Kostera-Pruszczyk, Anna
Servais, Laurent
Gerber, Marianne
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Scalco, Renata S.
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[5] SUNFISH Part 2: Efficacy and safety of risdiplam (RG7916) in patients with Type 2 or non-ambulant Type 3 spinal muscular atrophy (SMA)
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EUROPEAN JOURNAL OF NEUROLOGY, 2020, 27 : 869 - 869
[6] SUNFISH Part 2: Efficacy and Safety of Risdiplam (RG7916) in Patients with Type 2 or Non-Ambulant Type 3 Spinal Muscular Atrophy (SMA)
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NEUROLOGY, 2020, 94 (15)
[7] SUNFISH Part 2: 24-month efficacy and safety of risdiplam in patients with Type 2 or non-ambulant Type 3 spinal muscular atrophy (SMA)
Nascimento, A.
Day, J.
Deconinck, N.
Mazzone, E.
Oskoui, M.
Saito, K.
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Baranello, G.
Boespflug-Tanguy, O.
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Kostera-Pruszczyk, A.
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NEUROMUSCULAR DISORDERS, 2021, 31 : S136 - S136
[8] 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.
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Yeung, W. Yin
Mercuri, E.
EUROPEAN JOURNAL OF NEUROLOGY, 2021, 28 : 187 - 188
[9] Impact of nusinersen on respiratory progression in paediatric patients with spinal muscular atrophy type 2 and non-ambulant type 3
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Main, M.
Pane, M.
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D'Amico, A.
Bruno, C.
De Vivo, D.
Darras, B.
Day, J.
Baranello, G.
Sansona, V.
Bertini, E.
Finkel, R.
Mercuri, E.
Muntoni, F.
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[10] FIREFISH Parts 1 and 2: 4-year efficacy and safety of risdiplam in Type 1 spinal muscular atrophy (SMA)
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Day, J.
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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

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