At a glance: the largest Niemann-Pick type C1 cohort with 602 patients diagnosed over 15 years

Overview of attention for article published in European Journal of Human Genetics, July 2023

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6

The most recent citing publications are shown below. View all 6 publications that cite this research output on Dimensions.

Chapter 36 The Niemann–Pick diseases

Book chapter (January 2025)

Advances in research on potential therapeutic approaches for Niemann-Pick C1 disease

Article in Frontiers in Pharmacology (August 2024)

Optimization of systemic AAV9 gene therapy in Niemann-Pick disease type C1 mice

Preprint in bioRxiv (June 2024)

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