Tag: rare diseases

22-CHP-2790658-CQD–Hanna—Hunter-syndrome

Lysosomal Storage Diseases in Children: Considerations for Care

Physicians shares insights about screening, supportive care and potential therapies in development

18-NEU-765-Sideroblastic-Anemia-650×450

Pioneering Study Aims to Bring Light to the Shadows Around Pearson Syndrome

First-ever natural history study of a devastating mitochondrial disease

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