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Clinical Trials
Managed Access
MAA Resources for patients and physicians
Research
Publications list
Communications
Publications list
Advice
News
Mortality in patients with alpha‑mannosidosis: a review of patients’ data and the literature
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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
Expert guidance on Covid-19 for patients