Booklets
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Pompe Disease Treatments
POMPE | FEB 2026
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Late Onset Pompe Disease booklet and appointment sheet
POMPE | FEB 2026
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Infantile Onset Pompe Disease booklet and appointment sheet
POMPE | FEB 2026
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MPS Matters 2025. Insights and IMPACT
MPS | NOV 2025
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Fabry Matters Insights Preview
FABRY | 2024
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Living well with Fabry: Shared decision-making toolkit
FABRY | AUG 2024
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How physiotherapy can help with the physical symptoms of MPS III Sanfilippo
MPS III | MAR 2023
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My Fabry Treatment
FABRY | AUG 2022
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Expert guidance on Covid-19 for patients
LYSOSOMAL STORAGE DISEASES | APR 2021
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Focus on Fabry. Fabry treatment update
FABRY | OCT 2020
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Brineura treatment for CLN2 disease – The Managed Access Agreement Guide
CLN2 (BATTEN) | JUL 2020
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Vimizim treatment for Morquio A – The Managed Access Agreement
MPS IVA | JUN 2019