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Cerliponase Alfa for the Treatment of Atypical Phenotypes of CLN2 Disease: A Retrospective Case Series
BACKGROUND: The classic phenotype of CLN2 disease (neuronal ceroid lipofuscinosis type 2) typically manifests between ages 2 and 4 years with a predictable clinical course marked by epilepsy, language developmental delay, and rapid psychomotor decline. Atypical phenotypes exhibit variable time of on...
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| Veröffentlicht in: | J Child Neurol |
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| Hauptverfasser: | , , , , , , , , , , , , , , , |
| Format: | Artigo |
| Sprache: | Inglês |
| Veröffentlicht: |
SAGE Publications
2020
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| Schlagworte: | |
| Online Zugang: | https://ncbi.nlm.nih.gov/pmc/articles/PMC8027928/ https://ncbi.nlm.nih.gov/pubmed/33356800 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1177/0883073820977997 |
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