Long‐term benefit of enzyme replacement therapy with alglucosidase alfa in adults with Pompe disease: Prospective analysis from the French Pompe Registry
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DOI: 10.1002/jimd.12272
Abstract: Despite a wide clinical spectrum, the adult form of Pompe disease is the most common one, and represents more than 90% of diagnosed patients in France. Since the marketing of enzyme replacement therapy (alglucosidase alfa,… read more here.
Keywords: replacement therapy; therapy alglucosidase; enzyme replacement; pompe ... See more keywords