Long-term safety outcomes and patient preferences for home-based intravenous enzyme replacement therapy (ERT) in Pompe disease and Mucopolysaccharidosis Type I (MPS-I): final results of two-year observation.

ABSTRACT:

SUBMITTER: Toscano A 

PROVIDER: S-EPMC12752199 | biostudies-literature | 2025 Nov

REPOSITORIES: biostudies-literature