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

[21] Efficacy and safety of nusinersen treated adult patients with spinal muscular atrophy (SMA) types 2-3-4
De Wel, B.
Claeys, K.
NEUROMUSCULAR DISORDERS, 2020, 30 : S128 - S128
[22] 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)
[23] 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)
[24] FIREFISH Part 2: Efficacy and safety of risdiplam (RG7916) in infants with Type 1 spinal muscular atrophy (SMA)
Baranello, Giovanni
Servais, Laurent
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.
EUROPEAN RESPIRATORY JOURNAL, 2020, 56
[25] 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
[26] RAINBOWFISH: Preliminary efficacy and safety data in risdiplam-treated infants with presymptomatic spinal muscular atrophy (SMA)
Finkel, R.
Farrar, M.
Vlodavets, D.
Zanoteli, E.
Al-Muhaizea, M.
Nelson, L.
Prufer, A.
Servais, L.
Wang, Y.
Fisher, C.
Gerber, M.
Gorni, K.
Kletzl, H.
Palfreeman, L.
Scalco, R.
Bertini, E.
NEUROMUSCULAR DISORDERS, 2022, 32 : S85 - S86
[27] RAINBOWFISH: Primary efficacy and safety data in risdiplam-treated infants with presymptomatic spinal muscular atrophy (SMA)
Finkel, R.
Farrar, M.
Servais, L.
Vlodavets, D.
Zanoteli, E.
Al-Muhaizea, M.
Prufer, A.
Nelson, L.
Fischer, C.
Gerber, M.
Gorni, K.
Kletzl, H.
Palfreeman, L.
Gaki, E.
Fontoura, P.
Bertini, E.
NEUROMUSCULAR DISORDERS, 2023, 33 : S88 - S88
[28] 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
[29] SUNFISH Part 1: 24-month safety and exploratory outcomes of risdiplam (RG7916) treatment in patients with Type 2 or 3 spinal muscular atrophy (SMA)
Day, J.
Baranello, G.
Boespflug-Tanguy, O.
Borell, S.
Goemans, N.
Kirschner, J.
Masson, R.
Pera, M.
Servais, L.
Fuhrer, S.
Gerber, M.
Gorni, K.
Kletzl, H.
Martin, C.
Scalco, R.
Staunton, H.
Yeung, W.
Mercuri, E.
NEUROMUSCULAR DISORDERS, 2020, 30 : S123 - S123
[30] Safety and Efficacy of Apitegromab in Patients With Spinal Muscular Atrophy Types 2 and 3
Crawford, Thomas O.
Darras, Basil T.
Day, John W.
Young, Sally Dunaway
Duong, Tina
Nelson, Leslie L.
Barrett, Doreen
Song, Guochen
Bilic, Sanela
Cote, Shaun
Sadanowicz, Mara
Iarrobino, Ryan
Xu, Tiina J.
O'Neil, Janet
Rossello, Jose
Place, Amy
Kertesz, Nathalie
Nomikos, George
Chyung, Yung
NEUROLOGY, 2024, 102 (05) : e209151

← 1 2 3 4 5 →