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Clinical Trials
Managed Access
MAA Resources for patients and physicians
Evidence
Publications list
Research
Publications list
Communications
Publications list
Advice
Disease progression of Alpha-mannosidosis – A UK natural history survey – manuscript
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Understanding Fabry in Families Study – the availability of pedigree testing, genetic counselling and understanding of inheritance across Fabry International Network countries
Pathway to diagnosis and burden of illness in MPS VII – a European caregiver survey