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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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Disease Burden and Unmet Needs: Results from a New Survey in Adult Patients Receiving
Enzyme Replacement Therapy for Pompe Disease in the United Kingdom
Supporting adults living with
mucopolysaccharide (MPS) diseases:
Understanding current experiences and future challenges