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Fig. 4 | Orphanet Journal of Rare Diseases

Fig. 4

From: Impact of long-term elosulfase alfa treatment on clinical and patient-reported outcomes in patients with mucopolysaccharidosis type IVA: results from a Managed Access Agreement in England

Fig. 4

Change from baseline in MPS-HAQ domains over years 1, 2 and 3 of the MAA compared to change from baseline over 1 and 2 years reported in the MOR-001 natural history study. For ex-trial patients, pre-treatment baseline was used. Reductions in MPS-HAQ scores represent improvements. Error bars represent standard error

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Orphanet Journal of Rare Diseases

ISSN: 1750-1172