SUNFISH Parts 1 and 2: 4-year Efficacy and Safety Data of Risdiplam in Types 2 and 3 Spinal Muscular Atrophy (SMA)

被引：1

作者：

Servais, Laurent ^{[1
,2
,3
]}

Oskoui, Maryam ^{[4
]}

Day, John ^{[5
]}

Deconinck, Nicholas ^{[6
,7
]}

Mazzone, Elena ^{[8
]}

Nascimento, Andres ^{[9
]}

Saito, Kyoko ^{[10
]}

Vuillerot, Carole ^{[11
,12
]}

Baranello, Giovanni ^{[13
,14
,15
]}

Boespflug-Tanguy, Odile ^{[16
]}

Goemans, Nathalie ^{[17
]}

Kirschner, Janbernd ^{[18
]}

Kostera-Pruszczyk, Anna ^{[19
]}

Braid, Jessica ^{[20
]}

Papp, Gergely ^{[21
]}

Gorni, Ksenija ^{[22
]}

Martin, Carmen ^{[20
]}

Scalco, Renata ^{[23
]}

Yeung, Wai Yin ^{[20
]}

Mercuri, Eugenio ^{[21
]}

机构：

[1] Hop Armand Trousseau, Mot Inst Myol AP HP, Paris, France

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

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

[4] McGill Univ, Dept Pediat & Neurol Neurosurg, Montreal, PQ, Canada

[5] Stanford Univ, Dept Neurol, Palo Alto, CA USA

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

[7] UZ Gent, Neuromuscular Ctr, Ghent, Belgium

[8] Fondazione Policlinico Gemelli IRCCS, Cathol Univ & Nemo Pediat, Pediat Neurol Inst, Rome, Italy

[9] Fdn Sant Joan Deu, Hosp Sant Joan Deu, Neuromuscular Unit,Neuropaediat Dept, CIBERER,ISCIII, Barcelona, Spain

[10] Tokyo Womens Med Univ, Inst Med Genet, Tokyo, Japan

[11] CHU Lyon, Dept Pediat Phys Med & Rehabil, Hop Mere Enfant, Lyon, France

[12] Univ Lyon, CNRS UMR 5310 INSERM U1217, Neuromyogen Inst, Lyon, France

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

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

[15] Fondazione IRCCS Ist Neurol Carlo Besta, Dev Neurol Unit, Milan, Italy

[16] Hop Armand Trousseau, Mot Inst Myol AP HP, Paris, France

[17] Univ Paris Cite, UMR 1141, Neurodiderot, Paris, France

[18] Univ Hosp Leuven, Dept Paediat & Child Neurol, Neuromuscular Reference Ctr, Leuven, Belgium

[19] Univ Freiburg, Dept Neuropediat & Muscle Disorders, Med Ctr, Freiburg, Germany

[20] Med Univ Warsaw, Dept Neurol, Warsaw, Poland

[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

来源：

NEUROLOGY | 2023年 / 100卷 / 17期

关键词：

D O I：

10.1212/WNL.0000000000203570

中图分类号：

R74 [神经病学与精神病学];

学科分类号：

摘要：

S34.009

引用

页数：7

共 50 条

[1] Sunfish parts 1 and 2: 4-year efficacy and safety data of risdiplam in types 2 and 3 spinal muscular atrophy (SMA)
Oskoui, Maryam
Deconinck, Nicolas
Mazzone, Elena S.
Nascimento, Andres
Day, John W.
Saito, Kayoko
Vuillerot, Carole
Baranello, Giovanni
Boespflug-Tanguy, Odile
Goemans, Nathalie
Kirschnero, Janbernd
Kostera-Pruszczyk, Anna
Servais, Laurent
Braid, Jessica
Gerber, Marianne
Gorni, Ksenija
Martin, Carmen
Yeung, Wai Yin
Scalco, Renata S.
Mercuri, Eugenio
JOURNAL OF THE NEUROLOGICAL SCIENCES, 2023, 455
[2] SUNFISH Parts 1 and 2: 4-year efficacy and safety data of risdiplam in Types 2 and 3 spinal muscular atrophy (SMA)
Kostera-Pruszczyk, A.
Day, J.
Deconinck, N.
Mazzone, E.
Nascimento, A.
Oskoui, M.
Saito, K.
Vuillerot, C.
Baranello, G.
Boespflug-Tanguy, O.
Goemans, N.
Kirschner, J.
Servais, L.
Braid, J.
Gerber, M.
Gorni, K.
Martin, C.
Yeung, W.
Scalco, R.
Mercuri, E.
EUROPEAN JOURNAL OF NEUROLOGY, 2023, 30 : 194 - 194
[3] SUNFISH parts 1 and 2: 3-year efficacy and safety of risdiplam in types 2 and 3 spinal muscular atrophy (SMA)
Day, J.
Deconinck, N.
Mazzone, E.
Nascimento, A.
Oskoui, M.
Saito, K.
Vuillerot, C.
Baranello, G.
Boespflug-Tanguy, O.
Goemans, N.
Kirschner, J.
Kostera-Pruszczyk, A.
Servais, L.
Braid, J.
Gerber, M.
Gorni, K.
Martin, C.
Scalco, R.
Yeung, W.
Mercuri, E.
NEUROMUSCULAR DISORDERS, 2022, 32 : S89 - S90
[4] 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
[5] SUNFISH: 3-year efficacy and safety of risdiplam in Types 2 and 3 spinal muscular atrophy
Goemans, N.
Day, J.
Deconinck, N.
Mazzone, E.
Nascimento, A.
Oskoui, M.
Saito, K.
Vuillerot, C.
Baranello, G.
Boespflug-Tanguy, O.
Kirschner, J.
Kostera-Pruszczyk, A.
Servais, L.
Braid, J.
Gerber, M.
Gorni, K.
Martin, C.
Scalco, R.
Yeung, W.
Mercuri, E.
EUROPEAN JOURNAL OF NEUROLOGY, 2022, 29 : 278 - 278
[6] SUNFISH: 3-year Efficacy and Safety of Risdiplam in Types 2 and 3 SMA
Day, John W.
Deconinck, Nicolas
Kirschner, Janbernd
Mazzone, Elena S.
Nascimento, Andres
Oskoui, Maryam
Saito, Kyoko
Vuillerot, Carole
Baranello, Giovanni
Boespflug-Tanguy, Odile
Goemans, Nathalie
Kostera-Pruszczyk, Anna
Servais, Laurent
Braid, Jessica
Gerber, Marianne
Gorni, Ksenija
Martin, Carmen
Scala, Renata S.
Yeung, Wai Yin
Mercuri, Eugenio
NEUROLOGY, 2022, 98 (18)
[7] SUNFISH 3-YEAR EFFICACY AND SAFETY OF RISDIPLAM IN TYPES 2 AND 3 SMA
Servais, Laurent
Day, John W.
Mazzone, Elena S.
Oskoui, Maryam
Baranello, Giovanni
Gerber, Marianne
Martin, Carmen
McIver, Tammy
Yeung, Wai Yin
Mercuri, Eugenio
JOURNAL OF NEUROLOGY NEUROSURGERY AND PSYCHIATRY, 2022, 93 (09):
[8] 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
[9] 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)
[10] 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

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