Clinical and molecular characterization of Korean children with infantile and late-onset Pompe disease: 10 years of experience with enzyme replacement therapy at a single center.

Conclusion: Early identification of PD is very important, as ERT is effective for treatment. Patients with IOPD should be considered as candidates for clinical trials of new drugs in the future, as this study indicates the effectiveness of ERT in IOPD. PMID: 30360039 [PubMed - as supplied by publisher]
Source: Korean Journal of Pediatrics - Category: Pediatrics Tags: Korean J Pediatr Source Type: research