Improved Enzyme Replacement Therapy with Cipaglucosidase Alfa/Miglustat in Infantile Pompe Disease

Pompe disease is a lysosomal storage disorder with impaired glycogen degradation caused by a deficiency of the enzyme acid α-glucosidase (GAA). Children with the severe infantile form do not survive beyond the first year of life without treatment. Since 2006, enzyme replacement therapy (ERT) with Al...

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Detalles Bibliográficos
Autores principales: Fiege, Lina, Duran, Ibrahim, Marquardt, Thorsten
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2023
Materias:
Case Report
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10537092/
https://www.ncbi.nlm.nih.gov/pubmed/37765007
http://dx.doi.org/10.3390/ph16091199

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10537092/
https://www.ncbi.nlm.nih.gov/pubmed/37765007
http://dx.doi.org/10.3390/ph16091199

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