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

• Үндсэн зохиолчид: Vincenza Gragnaniello (Зохиогч), Federica Deodato (Зохиогч), Serena Gasperini (Зохиогч), Maria Alice Donati (Зохиогч), Clementina Canessa (Зохиогч), Simona Fecarotta (Зохиогч), Antonia Pascarella (Зохиогч), Giuseppe Spadaro (Зохиогч), Daniela Concolino (Зохиогч), Alberto Burlina (Зохиогч), Giancarlo Parenti (Зохиогч), Pietro Strisciuglio (Зохиогч), Agata Fiumara (Зохиогч), Roberto Della Casa (Зохиогч)
• Формат: Ном
• Хэвлэсэн: BMC, 2022-03-01T00:00:00Z.
• Нөхцлүүд: article
• Онлайн хандалт: 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...