Cipaglucosidase alfa-atga: Unveiling new horizons in Pompe disease therapy
Pompe disease is a lysosomal storage disease characterized by impaired glycogen breakdown due to an acid α-glucosidase (GAA) enzyme deficiency. Without therapy, children with the severe infantile form do not survive past their first year of life. POMBILITI which is intended to treat late-onset Pompe...
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| Hauptverfasser: | , , , , , , , |
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| Format: | Artigo |
| Sprache: | Inglês |
| Veröffentlicht: |
Elsevier
2024-06-01
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| Schriftenreihe: | Health Sciences Review |
| Schlagworte: | |
| Online-Zugang: | http://www.sciencedirect.com/science/article/pii/S2772632024000138 |
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