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Refinado por: Nome da Publicação: Neuromuscular Disorders : Nmd remover nível superior: Revistas revisadas por pares remover
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1
P46 ENVOL, a Phase 2, open-label trial evaluating the safety and expression of delandistrogene moxeparvovec in Duchenne muscular dystrophy: study design
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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 design

Mercuri, 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.V

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2
P.19 Updated demographics and safety data from patients with nonsense mutation Duchenne muscular dystrophy receiving ataluren in the STRIDE Registry
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P.19 Updated demographics and safety data from patients with nonsense mutation Duchenne muscular dystrophy receiving ataluren in the STRIDE Registry

Muntoni, 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]

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3
P196 Age at loss of ambulation in patients with DMD from the STRIDE registry and the CINRG natural history study: a matched cohort analysis
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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 analysis

Mercuri, 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.V

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4
P197 Pulmonary function in patients with Duchenne muscular dystrophy from the STRIDE registry and CINRG natural history study: a matched cohort analysis
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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 analysis

Tulinius, 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.V

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5
P198 Updated demographics and safety data from patients with nonsense mutation Duchenne muscular dystrophy receiving ataluren in the STRIDE registry
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P198 Updated demographics and safety data from patients with nonsense mutation Duchenne muscular dystrophy receiving ataluren in the STRIDE registry

Muntoni, 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.V

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6
P.22 Age at loss of ambulation in patients with DMD from the STRIDE registry and the CINRG natural history study: A matched cohort analysis
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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 analysis

Mercuri, 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]

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7
P.23 Pulmonary function in patients with Duchenne muscular dystrophy from the STRIDE Registry and CINRG Natural History Study: a matched cohort analysis
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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 analysis

Tulinius, 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]

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8
MITOCHONDRIAL DISEASES
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MITOCHONDRIAL DISEASES

Carrera, 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]

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9
MITOCHONDRIAL DISEASES: EP.214 Early onset hereditary neuropathies related to mitochondrial dynamics in pediatric patients
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MITOCHONDRIAL DISEASES: EP.214 Early onset hereditary neuropathies related to mitochondrial dynamics in pediatric patients

Carrera, 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.V

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10
DMD & BMD – CLINICAL
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DMD & BMD – CLINICAL

Muntoni, 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]

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