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The IMPACT survey: a mixed methods study to understand the experience of children, adolescents and adults with osteogenesis imperfecta and their caregivers.
Abbreviated Antiplatelet Therapy After Coronary Stenting in Patients With Myocardial Infarction at High Bleeding Risk.
🥇 Ensaio randomizadoDirect and indirect costs and cost-driving factors in adults with tuberous sclerosis complex: a multicenter cohort study and a review of the literature.
📖 RevisãoEconomic impact of screening for X-linked Adrenoleukodystrophy within a newborn blood spot screening programme.
Cost-effectiveness of enzyme replacement therapy for type 1 Gaucher disease.