Safety and efficacy of olesoxime in patients with type 2 or non-ambulatory type 3 spinal muscular atrophy: a randomised, double-blind, placebo-controlled phase 2 trial

Enrico Bertini; Eric Dessaud; Eugenio Mercuri; Francesco Muntoni; Janbernd Kirschner; Carol Reid; Anna Lusakowska; Giacomo P Comi; Jean-Marie Cuisset; Jean-Louis Abitbol; Bruno Scherrer; Patricia Sanwald Ducray; Jeppe Buchbjerg; Eduardo Vianna; W Ludo van der Pol; Carole Vuillerot; Thomas Blaettler; Paulo Fontoura; Carole André; Claudio Bruno; Brigitte Chabrol; Nicolas Deconinck; Brigitte Estournet; Stephanie Fontaine-Carbonnel; Nathalie Goemans; Ksenija Gorni; Alessandra Govoni; Michela Guglieri; Hanns Lochmuller; Francesca Magri; Michele Mayer; Wolfgang Müller-Felber; François Rivier; Helen Roper; Ulrike Schara; Mariacristina Scoto; Leonard van den Berg; Giuseppe Vita; Maggie C Walter

doi:10.1016/s1474-4422(17)30085-6

Safety and efficacy of olesoxime in patients with type 2 or non-ambulatory type 3 spinal muscular atrophy: a randomised, double-blind, placebo-controlled phase 2 trial

The Lancet Neurology ◽

10.1016/s1474-4422(17)30085-6 ◽

2017 ◽

Vol 16 (7) ◽

pp. 513-522 ◽

Cited By ~ 55

Author(s):

Enrico Bertini ◽

Eric Dessaud ◽

Eugenio Mercuri ◽

Francesco Muntoni ◽

Janbernd Kirschner ◽

...

Keyword(s):

Spinal Muscular Atrophy ◽

Muscular Atrophy ◽

Phase 2 ◽

Double Blind ◽

Safety And Efficacy ◽

Double Blind Placebo ◽

Phase 2 Trial ◽

Type 3

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A multinational, randomized, double-blind, placebo-controlled Phase 2 study to assess safety and efficacy of olesoxime in Type 2 or non-ambulatory Type 3 spinal muscular atrophy

Neuromuscular Disorders ◽

10.1016/s0960-8966(17)30304-8 ◽

2017 ◽

Vol 27 ◽

pp. S29

Author(s):

J. Buchbjerg ◽

E. Bertini ◽

E. Dessaud ◽

E. Mercuri ◽

F. Muntoni ◽

...

Keyword(s):

Spinal Muscular Atrophy ◽

Muscular Atrophy ◽

Phase 2 ◽

Double Blind ◽

Safety And Efficacy ◽

Double Blind Placebo ◽

Phase 2 Study ◽

Type 3

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Olesoxime in patients with type 2 or non-ambulatory type 3 Spinal muscular atrophy: a placebo-controlled phase 2 trial including a long-term, open-label follow-up study

Neuromuscular Disorders ◽

10.1016/j.nmd.2017.06.417 ◽

2017 ◽

Vol 27 ◽

pp. S209-S210

Author(s):

F. Muntoni ◽

J. Buchbjerg ◽

E. Bertini ◽

E. Dessaud ◽

E. Mercuri ◽

...

Keyword(s):

Spinal Muscular Atrophy ◽

Muscular Atrophy ◽

Phase 2 ◽

Open Label ◽

Follow Up Study ◽

Phase 2 Trial ◽

Type 3

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Safety and efficacy of once-daily risdiplam in type 2 and non-ambulant type 3 spinal muscular atrophy (SUNFISH part 2): a phase 3, double-blind, randomised, placebo-controlled trial

The Lancet Neurology ◽

10.1016/s1474-4422(21)00367-7 ◽

2022 ◽

Vol 21 (1) ◽

pp. 42-52 ◽

Cited By ~ 1

Author(s):

Eugenio Mercuri ◽

Nicolas Deconinck ◽

Elena S Mazzone ◽

Andres Nascimento ◽

Maryam Oskoui ◽

...

Keyword(s):

Spinal Muscular Atrophy ◽

Muscular Atrophy ◽

Controlled Trial ◽

Phase 3 ◽

Double Blind ◽

Once Daily ◽

Safety And Efficacy ◽

Type 3

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Safety and efficacy of ALD403, an antibody to calcitonin gene-related peptide, for the prevention of frequent episodic migraine: a randomised, double-blind, placebo-controlled, exploratory phase 2 trial

The Lancet Neurology ◽

10.1016/s1474-4422(14)70209-1 ◽

2014 ◽

Vol 13 (11) ◽

pp. 1100-1107 ◽

Cited By ~ 225

Author(s):

David W Dodick ◽

Peter J Goadsby ◽

Stephen D Silberstein ◽

Richard B Lipton ◽

Jes Olesen ◽

...

Keyword(s):

Episodic Migraine ◽

Calcitonin Gene Related Peptide ◽

Phase 2 ◽

Double Blind ◽

Safety And Efficacy ◽

Double Blind Placebo ◽

Related Peptide ◽

Phase 2 Trial ◽

Calcitonin Gene

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SAT0575 Safety and efficacy of lutikizumab (ABT-981), an anti–interleukin-1 alpha/beta dual variable domain (DVD) immunoglobulin, in subjects with knee osteoarthritis: results from the randomised, double-blind, placebo-controlled, parallel-group phase 2 trial

10.1136/annrheumdis-2018-eular.2727 ◽

2018 ◽

Author(s):

R. Fleischmann ◽

H. Bliddal ◽

F. Blanco ◽

T. Schnitzer ◽

C. Peterfy ◽

...

Keyword(s):

Knee Osteoarthritis ◽

Interleukin 1 ◽

Dual Variable ◽

Phase 2 ◽

Double Blind ◽

Safety And Efficacy ◽

Double Blind Placebo ◽

Phase 2 Trial ◽

Parallel Group ◽

Alpha Beta

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Safety and efficacy of amiselimod in relapsing multiple sclerosis (MOMENTUM): a randomised, double-blind, placebo-controlled phase 2 trial

The Lancet Neurology ◽

10.1016/s1474-4422(16)30192-2 ◽

2016 ◽

Vol 15 (11) ◽

pp. 1148-1159 ◽

Cited By ~ 28

Author(s):

Ludwig Kappos ◽

Douglas L Arnold ◽

Amit Bar-Or ◽

John Camm ◽

Tobias Derfuss ◽

...

Keyword(s):

Multiple Sclerosis ◽

Phase 2 ◽

Double Blind ◽

Safety And Efficacy ◽

Double Blind Placebo ◽

Phase 2 Trial ◽

Relapsing Multiple Sclerosis

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Faculty Opinions recommendation of Safety and efficacy of ALD403, an antibody to calcitonin gene-related peptide, for the prevention of frequent episodic migraine: a randomised, double-blind, placebo-controlled, exploratory phase 2 trial.

Faculty Opinions – Post-Publication Peer Review of the Biomedical Literature ◽

10.3410/f.720592850.793525445 ◽

2016 ◽

Author(s):

Mark Obermann ◽

Dagny Holle

Keyword(s):

Episodic Migraine ◽

Calcitonin Gene Related Peptide ◽

Phase 2 ◽

Double Blind ◽

Safety And Efficacy ◽

Double Blind Placebo ◽

Related Peptide ◽

Phase 2 Trial ◽

Calcitonin Gene

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Safety and efficacy of intravenous glyburide on brain swelling after large hemispheric infarction (GAMES-RP): a randomised, double-blind, placebo-controlled phase 2 trial

The Lancet Neurology ◽

10.1016/s1474-4422(16)30196-x ◽

2016 ◽

Vol 15 (11) ◽

pp. 1160-1169 ◽

Cited By ~ 99

Author(s):

Kevin N Sheth ◽

Jordan J Elm ◽

Bradley J Molyneaux ◽

Holly Hinson ◽

Lauren A Beslow ◽

...

Keyword(s):

Phase 2 ◽

Brain Swelling ◽

Double Blind ◽

Safety And Efficacy ◽

Double Blind Placebo ◽

Phase 2 Trial ◽

Hemispheric Infarction

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Safety and efficacy of AMG 334 for prevention of episodic migraine: a randomised, double-blind, placebo-controlled, phase 2 trial

The Lancet Neurology ◽

10.1016/s1474-4422(16)00019-3 ◽

2016 ◽

Vol 15 (4) ◽

pp. 382-390 ◽

Cited By ~ 187

Author(s):

Hong Sun ◽

David W Dodick ◽

Stephen Silberstein ◽

Peter J Goadsby ◽

Uwe Reuter ◽

...

Keyword(s):

Episodic Migraine ◽

Phase 2 ◽

Double Blind ◽

Safety And Efficacy ◽

Double Blind Placebo ◽

Phase 2 Trial

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Safety and efficacy of ozanezumab in patients with amyotrophic lateral sclerosis: a randomised, double-blind, placebo-controlled, phase 2 trial

The Lancet Neurology ◽

10.1016/s1474-4422(16)30399-4 ◽

2017 ◽

Vol 16 (3) ◽

pp. 208-216 ◽

Cited By ~ 26

Author(s):

Vincent Meininger ◽

Angela Genge ◽

Leonard H van den Berg ◽

Wim Robberecht ◽

Albert Ludolph ◽

...

Keyword(s):

Amyotrophic Lateral Sclerosis ◽

Phase 2 ◽

Double Blind ◽

Safety And Efficacy ◽

Double Blind Placebo ◽

Phase 2 Trial ◽

Lateral Sclerosis

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