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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 |
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Titel: |
Safety, tolerability and pharmacokinetics of eteplirsen in young boys aged 6–48 months with Duchenne muscular dystrophy amenable to exon 51 skipping |
Auteur: |
Mercuri, E. Seferian, A.M. Servais, L. Deconinck, N. Stevenson, H. Ni, X. Zhang, W. East, L. Yonren, S. Muntoni, F. |
Verschenen in: |
Neuromuscular disorders |
Paginering: |
Jaargang 33 () nr. 6 pagina's 476-483 |
Jaar: |
2023 |
Inhoud: |
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Uitgever: |
Published by Elsevier B.V. |
Bronbestand: |
Elektronische Wetenschappelijke Tijdschriften |
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