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Material Type: Artigo
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P46 ENVOL, a Phase 2, open-label trial evaluating the safety and expression of delandistrogene moxeparvovec in Duchenne muscular dystrophy: study designMercuri, E. ; Desguerre, I. ; Gangfuss, A. ; Servais, L. ; Nascimento, A. ; Zhang, B. ; Murphy, A. ; Reid, C. ; Wandel, C. ; Singh, T. ; Guridi, M. ; Muntoni, F.Neuromuscular disorders : NMD, 2023-10, Vol.33, p.S70-S70 [Periódico revisado por pares]Elsevier B.VTexto completo disponível |
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2 |
Material Type: Artigo
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P.19 Updated demographics and safety data from patients with nonsense mutation Duchenne muscular dystrophy receiving ataluren in the STRIDE RegistryMuntoni, F. ; Buccella, F. ; Desguerre, I. ; Kirschner, J. ; Osorio, A. Nascimento ; Tulinius, M. ; Johnson, S. ; Werner, C. ; Jiang, J. ; Li, J. ; Jia, J. ; Trifillis, P. ; Mercuri, E.Neuromuscular disorders : NMD, 2022-10, Vol.32, p.S51 [Periódico revisado por pares]Texto completo disponível |
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3 |
Material Type: Artigo
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P196 Age at loss of ambulation in patients with DMD from the STRIDE registry and the CINRG natural history study: a matched cohort analysisMercuri, E. ; Muntoni, F. ; Buccella, F. ; Desguerre, I. ; Kirschner, J. ; Osorio, A Nascimento ; Tulinius, M. ; de Resende, M. ; Morgenroth, L. ; Gordish-Dressman, H. ; Johnson, S. ; Werner, C. ; Anbu, B. ; Liu, E. ; Rajbhandari, R. ; Trifillis, P. ; McDonald, C.Neuromuscular disorders : NMD, 2023-10, Vol.33, p.S143-S143 [Periódico revisado por pares]Elsevier B.VTexto completo disponível |
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4 |
Material Type: Artigo
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P197 Pulmonary function in patients with Duchenne muscular dystrophy from the STRIDE registry and CINRG natural history study: a matched cohort analysisTulinius, M. ; Buccella, F. ; Desguerre, I. ; Kirschner, J. ; Mercuri, E. ; Muntoni, F. ; Osorio, A Nascimento ; de Resende, M. ; Morgenroth, L. ; Gordish-Dressman, H. ; Johnson, S. ; Werner, C. ; Anbu, B. ; Liu, E. ; Rajbhandari, R. ; Trifillis, P. ; McDonald, C.Neuromuscular disorders : NMD, 2023-10, Vol.33, p.S143-S143 [Periódico revisado por pares]Elsevier B.VTexto completo disponível |
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5 |
Material Type: Artigo
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P198 Updated demographics and safety data from patients with nonsense mutation Duchenne muscular dystrophy receiving ataluren in the STRIDE registryMuntoni, F. ; Buccella, F. ; Desguerre, I. ; Kirschner, J. ; Osorio, A Nascimento ; Tulinius, M. ; de Resende, M. ; Johnson, S. ; Werner, C. ; Anbu, B. ; Liu, E. ; Rajbhandari, R. ; Trifillis, P. ; Mercuri, E.Neuromuscular disorders : NMD, 2023-10, Vol.33, p.S143-S144 [Periódico revisado por pares]Elsevier B.VTexto completo disponível |
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6 |
Material Type: Artigo
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P.22 Age at loss of ambulation in patients with DMD from the STRIDE registry and the CINRG natural history study: A matched cohort analysisMercuri, E. ; Muntoni, F. ; Buccella, F. ; Desguerre, I. ; Kirschner, J. ; Osorio, A. Nascimento ; Tulinius, M. ; Morgenroth, L. ; Gordish-Dressman, H. ; Johnson, S. ; Werner, C. ; Jiang, J. ; Li, J. ; Jia, J. ; Trifillis, P. ; McDonald, C.Neuromuscular disorders : NMD, 2022-10, Vol.32, p.S52 [Periódico revisado por pares]Texto completo disponível |
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7 |
Material Type: Artigo
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P.23 Pulmonary function in patients with Duchenne muscular dystrophy from the STRIDE Registry and CINRG Natural History Study: a matched cohort analysisTulinius, M. ; Buccella, F. ; Desguerre, I. ; Kirschner, J. ; Mercuri, E. ; Muntoni, F. ; Osorio, A. Nascimento ; Morgenroth, L. ; Gordish-Dressman, H. ; Johnson, S. ; Werner, C. ; Jiang, J. ; Li, J. ; Jia, J. ; Trifillis, P. ; McDonald, C.Neuromuscular disorders : NMD, 2022-10, Vol.32, p.S52 [Periódico revisado por pares]Texto completo disponível |
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8 |
Material Type: Artigo
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MITOCHONDRIAL DISEASESCarrera, L. ; Cantarero, L. ; Nolasco, G. ; Pijuan, J. ; Natera, D. ; Estevez, B. ; Expósito, J. ; Colomer, J. ; Ortez, C. ; Palau, F. ; Hoenicka, J. ; Nascimento, A.Neuromuscular disorders : NMD, 2021-10, Vol.31, p.S114 [Periódico revisado por pares]Texto completo disponível |
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9 |
Material Type: Artigo
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MITOCHONDRIAL DISEASES: EP.214 Early onset hereditary neuropathies related to mitochondrial dynamics in pediatric patientsCarrera, L. ; Cantarero, L. ; Nolasco, G. ; Pijuan, J. ; Natera, D. ; Estevez, B. ; Expósito, J. ; Colomer, J. ; Ortez, C. ; Palau, F. ; Hoenicka, J. ; Nascimento, A.Neuromuscular disorders : NMD, 2021-10, Vol.31, p.S114-S114 [Periódico revisado por pares]Elsevier B.VTexto completo disponível |
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10 |
Material Type: Artigo
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DMD & BMD – CLINICALMuntoni, F. ; Mercuri, E. ; Buccella, F. ; Desguerre, I. ; Kirschner, J. ; Osorio, A. Nascimento ; Tulinius, M. ; Jiang, J. ; Kristensen, A. ; Johnson, S. ; Able, R. ; Trifillis, P. ; Santos, C.Neuromuscular disorders : NMD, 2020-10, Vol.30, p.S62 [Periódico revisado por pares]Texto completo disponível |