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Immune tolerance strategies in siblings with infantile Pompe disease — Advantages for a preemptive approach to high-sustained antibody titers

Enzyme replacement therapy (ERT) has led to a significant improvement in the clinical course of patients with infantile Pompe disease (IPD), an autosomal recessive glycogen storage disorder characterized by the deficiency in lysosomal acid α-glucosidase. A subset of IPD patients mounts a substantial...

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Detalles Bibliográficos
Autores principales:	Stenger, Elizabeth O., Kazi, Zoheb, Lisi, Emily, Gambello, Michael J., Kishnani, Priya
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Elsevier 2015
Materias:	SI:Therapy
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4497810/ https://www.ncbi.nlm.nih.gov/pubmed/26167453 http://dx.doi.org/10.1016/j.ymgmr.2015.05.004

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