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Clinical Trials
Managed Access
MAA Resources for patients and physicians
Research
Publications list
Communications
Publications list
Advice
News
Burden of illness in Sanfilippo disease (MPS III) – results from an international caregiver survey
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The educational journey of individuals with MPS IVA Morquio Disease
Patient organisations working in partnership to research the patient experience of rare diseases – the Sanfilippo (MPS III) survey