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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Union of pediatricians of Russia,
2014-09-01T00:00:00Z.
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