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

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

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Nanobioengineering - Institute for Bioengineering of Catalonia

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

The pathogenesis of Niemann–Pick type C disease: a role for

Gene Therapy & Molecular Biology Volume 11 Issue A by Niko

Drug Delivery and Translational Research期刊最新论文, 化学/材料

Full article: New therapies in the management of Niemann-Pick type

Gene therapy shows promise for treating Niemann-Pick disease type

Full article: New therapies in the management of Niemann-Pick type