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Clinical Trials
Managed Access
MAA Resources for patients and physicians
Research
Publications list
Communications
Publications list
Advice
News
Vimizim treatment for Morquio A – The Managed Access Agreement
A guide for patients and parents
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Multi-stakeholder engagement leading to access to treatment for MPS IVA (Morquio A) – a model for the ultra-rare disease community
Fabry Findings Issue 1