ALGLUCOSIDASE ALFA — A NEW STAGE IN THE THERAPY OF INFANTILE POMPE DISEASE

ALGLUCOSIDASE ALFA — A NEW STAGE IN THE THERAPY OF INFANTILE POMPE DISEASE

Pompe disease is a rare severe hereditary disease caused by excessive glycogen storage in organs and target tissues due to the acid α-glucosidase gene mutation. Infantile and adult Pompe disease is characterized by involvement of cardiovascular, respiratory and muscular systems in the pathological p...

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Bibliographic Details
Main Authors:	E. N. Basargina, E. N. Arkhipova, V. S. Ermolenko
Format:	Article
Language:	English
Published:	Paediatrician Publishers, LLC 2014-09-01
Series:	Pediatričeskaâ Farmakologiâ
Subjects:	pompe disease storage disease infantile disease enzyme replacement therapy
Online Access:	https://www.pedpharma.ru/jour/article/view/58

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