Impact of long-term elosulfase alfa treatment on respiratory function in patients with Morquio A syndrome

ConclusionsLong-term ERT is associated with sustained improvements in respiratory function in Morquio A. In younger patients ( ≤14 years), some improvement may be ascribed to growth. In older patients, other mechanisms, e.g., decreased glycosaminoglycan storage, are likely involved.
Source: Journal of Inherited Metabolic Disease - Category: Internal Medicine Source Type: research