Real-world data assessment of safety of home-based and hospital/outpatient-based laronidase enzyme replacement therapy for mucopolysaccharidosis I
oleh: Amanda Wilson, Raffaella M. Colzani, Ana Lorena Flores
| Format: | Article |
|---|---|
| Diterbitkan: | Elsevier 2021-06-01 |
Deskripsi
Subjek
Real-world evidence; enzyme replacement therapy; MPS I; Home-based infusion