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Clinical Trials
Managed Access
MAA Resources for patients and physicians
Research
Publications list
Communications
Publications list
Advice
News
Mortality in patients with Alpha-mannosidosis
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The importance of early diagnosis and views
on newborn screening in Metachromatic
Leukodystrophy
Impact of the COVID‑19 pandemic on access to the cerliponase alfa managed access agreement in England for CLN2 treatment