Summaries of latest research advances related to Niemann-Pick diseases, acid sphingomyelinase deficiency (ASMD) and Niemann-Pick type C disease (NPCD), based on selected peer-reviewed publications in scientific journals.
By Frank W. Pfrieger, PhD
Institute of Cellular and Integrative Neurosciences
Centre National de la Recherche Scientifique / University of Strasbourg
Strasbourg, France
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These summaries provide a glimpse into the diverse research efforts aimed at understanding and addressing NPCD and ASMD, shedding light on biomarkers, genetic modifiers, diagnosis, treatment approaches, and potential therapeutic targets.
Please feel free to distribute and forward this issue, and to send feedback to: fw-pfrieger@gmx.de or frank.pfrieger@unistra.fr
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