Cleary, M;
Davison, J;
Gould, R;
Geberhiwot, T;
Hughes, D;
Mercer, J;
Morrison, A;
... Stepien, KM; + view all
(2021)
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.
Orphanet Journal of Rare Diseases
, 16
, Article 38. 10.1186/s13023-021-01675-x.