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