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Refrigeration at the heart of biomedical innovation: the example of Libmeldy
A rare inherited disorder can now be cured with gene therapy. Libmeldy, an innovative drug, must be stored at a temperature of – 130°C.
Metachromatic leukodystrophy is a rare hereditary disease characterised by a change in a gene required for the production of the enzyme ARSA. This enzyme enables the breakdown of certain substances known as sulfatides. When not broken down, sulfatides damage organs and the nervous system.
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