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Autor:
Willy Berlier, Quitterie Barthe, Gaelle Campello, Yann Godfrin, Nathalie Guerin, Françoise Horand, Magali Cremel
Publikováno v:
International Journal of Pharmaceutics. 491:69-77
Pompe disease is a glycogen storage disease caused by acid α-glucosidase enzyme deficiency. Currently, the unique treatment is lifelong enzyme replacement therapy ERT with frequent intravenous administration of the recombinant analog alglucosidase-