Home-based enzyme replacement therapy in children and adults with Pompe disease; a prospective study
Abstract Background Pompe disease is a lysosomal storage disease treated with life-long enzyme replacement therapy (ERT). Home-based ERT has been provided in the Netherlands since 2008 because it diminishes the burden of treatment, increases patient flexibility and autonomy, and is thus a more patie...
| Автори: | , , , , , |
|---|---|
| Формат: | Стаття |
| Мова: | English |
| Опубліковано: |
BMC
2023-05-01
|
| Серія: | Orphanet Journal of Rare Diseases |
| Предмети: | |
| Онлайн доступ: | https://doi.org/10.1186/s13023-023-02715-4 |