Marta, with Niemann Pick C, fights for a cure with gene therapy - Fundación Columbus

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Last updated 07 abril 2025

Marta, with Niemann Pick C, fights for a cure with gene therapy - Fundación Columbus

Marta is a four and a half year old girl with Niemann Pick C. This is a rare neurodegenerative genetic disease

Selected Abstracts from the 12th Annual Meeting of the Clinical Immunology Society: 2021 Virtual Annual Meeting: Immune Deficiency and Dysregulation North American Conference. - Abstract - Europe PMC

delta – Columbus Children's Foundation

Selected Abstracts from the 12th Annual Meeting of the Clinical Immunology Society: 2021 Virtual Annual Meeting: Immune Deficiency and Dysregulation North American Conference

PDF) A Natural History of Floating Sargassum Species (Sargasso) from Mexico

Gene Therapy for Niemann-Pick Type a Diseases Moves Forward

Selected Abstracts from the 12th Annual Meeting of the Clinical Immunology Society: 2021 Virtual Annual Meeting: Immune Deficiency and Dysregulation North American Conference

3 Hispanic Families Facing Alzheimer's Disease

Gene therapy shows promise for treating Niemann-Pick disease type C1

Nancy Iafrate's second chance at life - UF Health

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