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...
| Main Authors: | , , |
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| Format: | Article |
| Language: | English |
| Published: |
Paediatrician Publishers, LLC
2014-09-01
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| Series: | Pediatričeskaâ Farmakologiâ |
| Subjects: | |
| Online Access: | https://www.pedpharma.ru/jour/article/view/58 |