Immune responses to alglucosidase in infantile Pompe disease: recommendations from an Italian pediatric expert panel

• Main Authors: Vincenza Gragnaniello (Author), Federica Deodato (Author), Serena Gasperini (Author), Maria Alice Donati (Author), Clementina Canessa (Author), Simona Fecarotta (Author), Antonia Pascarella (Author), Giuseppe Spadaro (Author), Daniela Concolino (Author), Alberto Burlina (Author), Giancarlo Parenti (Author), Pietro Strisciuglio (Author), Agata Fiumara (Author), Roberto Della Casa (Author)
• Format: Book
• Published: BMC, 2022-03-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

Abstract Background Classic infantile onset of Pompe disease (c-IOPD) leads to hypotonia and hypertrophic cardiomyopathy within the first days to weeks of life and, without treatment, patients die of cardiorespiratory failure in their first 1-2 years of life. Enzymatic replacement therapy (ERT) with...