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Sebelipase alfa for lysosomal acid lipase deficiency: 5‐year treatment experience from a phase 2 open‐label extension study |
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| Authors: | V&#x ra Malinov ,Manisha Balwani,Reena Sharma,Jean‐Baptiste Arnoux,John Kane,Chester B. Whitley,Sachin Marulkar,Florian Abel |
| Affiliation: | Vĕra Malinová,Manisha Balwani,Reena Sharma,Jean‐Baptiste Arnoux,John Kane,Chester B. Whitley,Sachin Marulkar,Florian Abel |
| Abstract: | |
| Keywords: | enzyme replacement therapy lipids liver lysosomal storage diseases transaminases |