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Clinical Trials
Managed Access
MAA Resources for patients and physicians
Research
Publications list
Communications
Publications list
Advice
News
Guidelines for the Li–Fraumeni and heritable TP53-related cancer syndromes
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The importance of psychological support for parents and caregivers of children with a rare disease at diagnosis
Elosulfase alfa in the treatment
of mucopolysaccharidosis type IVA: insights
from the first managed access agreement