The characterisation of pulmonary function in patients with mucopolysaccharidoses IVA: A longitudinal analysis

ConclusionsWhilst spirometry values showed a gradual decline across all groups, oximetry showed modest improvement in respiratory function. The amalgamation of FEV1, FVC [%predicted] and ODI 3% appeared predictive of changes in respiratory function in this study, suggestive as being composite endpoints for monitoring disease progression as well as guiding response to ERT in MPS IVA patients.
Source: Molecular Genetics and Metabolism Reports - Category: Genetics & Stem Cells Source Type: research