The SPARKLE registry: protocol for an international prospective cohort study in patients with alpha-mannosidosis
Abstract Background Alpha-mannosidosis is a lysosomal storage disorder caused by reduced enzymatic activity of alpha-mannosidase. SPARKLE is an alpha-mannosidosis registry intended to obtain long-term safety and effectiveness data on the use of velmanase alfa during routine clinical care in patients...
Збережено в:
| Автори: | , , , , , , , , |
|---|---|
| Формат: | Artigo |
| Мова: | Inglês |
| Опубліковано: |
BMC
2020-09-01
|
| Серія: | Orphanet Journal of Rare Diseases |
| Предмети: | |
| Онлайн доступ: | http://link.springer.com/article/10.1186/s13023-020-01549-8 |
| Теги: |
Немає тегів, Будьте першим, хто поставить тег для цього запису!
|