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
- Location
-
Deutsche Nationalbibliothek Frankfurt am Main
- ISSN
-
1750-1172
- Extent
-
Online-Ressource
- Language
-
Englisch
- Notes
-
online resource.
- Bibliographic citation
-
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
- Creator
-
Vijay, Suresh
Brassier, Anais
Ghosh, Arunabha
Fecarotta, Simona
Abel, Florian
Marulkar, Sachin
Jones, Simon A.
- Contributor
-
SpringerLink (Online service)
- DOI
-
10.1186/s13023-020-01577-4
- URN
-
urn:nbn:de:101:1-2021020723212325830536
- Rights
-
Open Access; Der Zugriff auf das Objekt ist unbeschränkt möglich.
- Last update
-
15.08.2025, 7:24 AM CEST
Data provider
This object is provided by:
Deutsche Nationalbibliothek. If you have any questions about the object, please contact the data provider.
Deutsche Nationalbibliothek. If you have any questions about the object, please contact the data provider.
Associated
- Vijay, Suresh
- Brassier, Anais
- Ghosh, Arunabha
- Fecarotta, Simona
- Abel, Florian
- Marulkar, Sachin
- Jones, Simon A.
- SpringerLink (Online service)
Other Objects (12)
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
Hepatosteatosis from Lysosomal Acid Lipase Deficiency
The rapidly evolving view of lysosomal storage diseases
Persistent dyslipidemia in treatment of lysosomal acid lipase deficiency
Long term substrate reduction therapy with ezetimibe alone or associated with statins in three adult patients with lysosomal acid lipase deficiency
Lysosomal acid lipase regulates VLDL synthesis and insulin sensitivity in mice
“Why them, why me, why us?” The experiences of parents of children with lysosomal acid lipase deficiency: an interpretative phenomenological analysis study
Fatty Liver and Systemic Atherosclerosis in a Young, Lean Patient: Rule Out Lysosomal Acid Lipase Deficiency
Early diagnosis of infantile-onset lysosomal acid lipase deficiency in the advent of available enzyme replacement therapy
Functional characterization of lysosomal interaction of Akt with VRK2
Withdrawal: Lipase maturation factor LMF1, membrane topology and interaction with lipase proteins in the endoplasmic reticulum
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
Hepatosteatosis from Lysosomal Acid Lipase Deficiency
The rapidly evolving view of lysosomal storage diseases
Persistent dyslipidemia in treatment of lysosomal acid lipase deficiency
Long term substrate reduction therapy with ezetimibe alone or associated with statins in three adult patients with lysosomal acid lipase deficiency
Lysosomal acid lipase regulates VLDL synthesis and insulin sensitivity in mice
“Why them, why me, why us?” The experiences of parents of children with lysosomal acid lipase deficiency: an interpretative phenomenological analysis study
Fatty Liver and Systemic Atherosclerosis in a Young, Lean Patient: Rule Out Lysosomal Acid Lipase Deficiency
Early diagnosis of infantile-onset lysosomal acid lipase deficiency in the advent of available enzyme replacement therapy
Functional characterization of lysosomal interaction of Akt with VRK2
Withdrawal: Lipase maturation factor LMF1, membrane topology and interaction with lipase proteins in the endoplasmic reticulum
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
Hepatosteatosis from Lysosomal Acid Lipase Deficiency
The rapidly evolving view of lysosomal storage diseases
Persistent dyslipidemia in treatment of lysosomal acid lipase deficiency
Long term substrate reduction therapy with ezetimibe alone or associated with statins in three adult patients with lysosomal acid lipase deficiency
Lysosomal acid lipase regulates VLDL synthesis and insulin sensitivity in mice
“Why them, why me, why us?” The experiences of parents of children with lysosomal acid lipase deficiency: an interpretative phenomenological analysis study
Fatty Liver and Systemic Atherosclerosis in a Young, Lean Patient: Rule Out Lysosomal Acid Lipase Deficiency
Early diagnosis of infantile-onset lysosomal acid lipase deficiency in the advent of available enzyme replacement therapy
Functional characterization of lysosomal interaction of Akt with VRK2