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1
Fonction des membres supérieurs chez les patients atteints de nmDMD d’après les données du registre STRIDE et de l’Étude 041, essai de phase 3, contrôlé contre placebo
Fonction des membres supérieurs chez les patients atteints de nmDMD d’après les données du registre STRIDE et de l’Étude 041, essai de phase 3, contrôlé contre placebo
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Fonction des membres supérieurs chez les patients atteints de nmDMD d’après les données du registre STRIDE et de l’Étude 041, essai de phase 3, contrôlé contre placebo

Damez-Werno, Diane ; Mcdonald, Craig ; Mercuri, Eugenio ; Muntoni, Francesco ; Gordish-Dressman, Heather ; Trifillis, Panayiota

Revue neurologique, 2023-04, Vol.179, p.S105-S106 [Periódico revisado por pares]

Elsevier Masson SAS

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2
Ataluren as an Agent for Therapeutic Nonsense Suppression
Ataluren as an Agent for Therapeutic Nonsense Suppression
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Ataluren as an Agent for Therapeutic Nonsense Suppression

Peltz, Stuart W ; Morsy, Manal ; Welch, Ellen M ; Jacobson, Allan

Annual review of medicine, 2013-01, Vol.64 (1), p.407-425 [Periódico revisado por pares]

United States: Annual Reviews

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3
Efficacy and safety of ataluren in patients with nonsense-mutation cystic fibrosis not receiving chronic inhaled aminoglycosides: The international, randomized, double-blind, placebo-controlled Ataluren Confirmatory Trial in Cystic Fibrosis (ACT CF)
Efficacy and safety of ataluren in patients with nonsense-mutation cystic fibrosis not receiving chronic inhaled aminoglycosides: The international, randomized, double-blind, placebo-controlled Ataluren Confirmatory Trial in Cystic Fibrosis (ACT CF)
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Efficacy and safety of ataluren in patients with nonsense-mutation cystic fibrosis not receiving chronic inhaled aminoglycosides: The international, randomized, double-blind, placebo-controlled Ataluren Confirmatory Trial in Cystic Fibrosis (ACT CF)

Konstan, M.W. ; VanDevanter, D.R. ; Rowe, S.M. ; Wilschanski, M. ; Kerem, E. ; Sermet-Gaudelus, I. ; DiMango, E. ; Melotti, P. ; McIntosh, J. ; De Boeck, K.

Journal of cystic fibrosis, 2020-07, Vol.19 (4), p.595-601 [Periódico revisado por pares]

Netherlands: Elsevier B.V

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4
Continuitiy of care with ataluren in Duchenne Muscular Dystrophy patients with nonsense mutations after loss of ambulation. Personal experience
Continuitiy of care with ataluren in Duchenne Muscular Dystrophy patients with nonsense mutations after loss of ambulation. Personal experience
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Continuitiy of care with ataluren in Duchenne Muscular Dystrophy patients with nonsense mutations after loss of ambulation. Personal experience

Spagnoli, Carlotta ; Adorisio, Rachele ; Bello, Luca ; D’Amico, Adele ; D’Angelo, Maria Grazia ; Pane, Marika ; Penzo, Martina ; Riguzzi, Pietro ; Sansone, Valeria ; Vianello, Andrea ; Fusco, Carlo

Acta myologica, 2023-12, Vol.42 (4), p.118-122 [Periódico revisado por pares]

Pacini Editore Srl

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5
Ataluren metabolism: Ataluren-O-1β-acyl glucuronide is a stable circulating metabolite in mouse, rat, dog and human
Ataluren metabolism: Ataluren-O-1β-acyl glucuronide is a stable circulating metabolite in mouse, rat, dog and human
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Ataluren metabolism: Ataluren-O-1β-acyl glucuronide is a stable circulating metabolite in mouse, rat, dog and human

Ma, Jiyuan ; Risher, Nicole ; Northcutt, Valerie ; Moon, Young-Choon ; Weetall, Marla ; Welch, Ellen ; Colacino, Joseph ; Almstead, Neil ; Kong, Ronald

Drug metabolism and pharmacokinetics, 2021-06, Vol.38, p.100393-100393, Article 100393 [Periódico revisado por pares]

England: Elsevier Ltd

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6
Synthesis and biological evaluation of 1,2,4-oxadiazole incorporated 1,2,3-triazole-pyrazole derivatives as anticancer agents
Synthesis and biological evaluation of 1,2,4-oxadiazole incorporated 1,2,3-triazole-pyrazole derivatives as anticancer agents
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Synthesis and biological evaluation of 1,2,4-oxadiazole incorporated 1,2,3-triazole-pyrazole derivatives as anticancer agents

G, Mohan ; Sridhar, Gattu ; Laxminarayana, E. ; Chary, M. Thirumala

Chemical Data Collections, 2021-08, Vol.34, p.100735, Article 100735 [Periódico revisado por pares]

Elsevier B.V

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7
Pharmacological advances for treatment in Duchenne muscular dystrophy
Pharmacological advances for treatment in Duchenne muscular dystrophy
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Pharmacological advances for treatment in Duchenne muscular dystrophy

Guiraud, Simon ; Davies, Kay E

Current opinion in pharmacology, 2017-06, Vol.34, p.36-48 [Periódico revisado por pares]

England: Elsevier Ltd

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8
Read-through approach for stop mutations in Duchenne muscular dystrophy. An update
Read-through approach for stop mutations in Duchenne muscular dystrophy. An update
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Read-through approach for stop mutations in Duchenne muscular dystrophy. An update

Politano, Luisa

Acta myologica, 2021-03, Vol.40 (1), p.43-50 [Periódico revisado por pares]

Pacini Editore Srl

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9
Impact of Disease-modifying Therapies on Respiratory Function in People with Neuromuscular Disorders
Impact of Disease-modifying Therapies on Respiratory Function in People with Neuromuscular Disorders
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Impact of Disease-modifying Therapies on Respiratory Function in People with Neuromuscular Disorders

Xiao, Lena ; Amin, Reshma

Sleep medicine clinics, 2024-06

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10
Safety, tolerability and pharmacokinetics of eteplirsen in young boys aged 6-48 months with Duchenne muscular dystrophy amenable to exon 51 skipping
Safety, tolerability and pharmacokinetics of eteplirsen in young boys aged 6-48 months with Duchenne muscular dystrophy amenable to exon 51 skipping
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Safety, tolerability and pharmacokinetics of eteplirsen in young boys aged 6-48 months with Duchenne muscular dystrophy amenable to exon 51 skipping

Mercuri, E ; Seferian, A. M ; Servais, L ; Deconinck, N ; Stevenson, H ; Ni, X ; Zhang, W ; East, L ; Yonren, S ; Muntoni, F ; Van Coster, Rudy ; Vanlander, Arnaud ; Study Group, 4658-102

2023

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