Long-term survival with sebelipase alfa enzyme replacement therapy in infants with rapidly progressive lysosomal acid lipase deficiency: final results from 2 open-label studies
- Standort
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Deutsche Nationalbibliothek Frankfurt am Main
- ISSN
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1750-1172
- Umfang
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Online-Ressource
- Sprache
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Englisch
- Anmerkungen
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online resource.
- Erschienen in
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Long-term survival with sebelipase alfa enzyme replacement therapy in infants with rapidly progressive lysosomal acid lipase deficiency: final results from 2 open-label studies ; volume:16 ; number:1 ; day:6 ; month:1 ; year:2021 ; pages:1-15 ; date:12.2021
Orphanet journal of rare diseases ; 16, Heft 1 (6.1.2021), 1-15, 12.2021
- Urheber
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Vijay, Suresh
Brassier, Anais
Ghosh, Arunabha
Fecarotta, Simona
Abel, Florian
Marulkar, Sachin
Jones, Simon A.
- Beteiligte Personen und Organisationen
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SpringerLink (Online service)
- DOI
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10.1186/s13023-020-01577-4
- URN
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urn:nbn:de:101:1-2021020723212325830536
- Rechteinformation
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Open Access; Der Zugriff auf das Objekt ist unbeschränkt möglich.
- Letzte Aktualisierung
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15.08.2025, 07:24 MESZ
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Beteiligte
- Vijay, Suresh
- Brassier, Anais
- Ghosh, Arunabha
- Fecarotta, Simona
- Abel, Florian
- Marulkar, Sachin
- Jones, Simon A.
- SpringerLink (Online service)
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Correction to: Long-term survival with sebelipase alfa enzyme replacement therapy in infants with rapidly progressive lysosomal acid lipase deficiency: final results from 2 open-label studies
Survival in infants treated with sebelipase Alfa for lysosomal acid lipase deficiency: an open-label, multicenter, dose-escalation study
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