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Libmeldy
autologous CD34+ cells encoding ARSA gene
Leukodystrophy, Metachromatic
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Authorised
Advanced therapy
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No active alerts
This medicine has no ongoing shortages, safety letters or recalls.
What it's used for
Libmeldy is indicated for the treatment of metachromatic leukodystrophy (MLD) characterized by biallelic mutations in the arysulfatase A (ARSA) gene leading to a reduction of the ARSA enzymatic activity: in children with late infantile or early juvenile forms, without clinical manifestations of the disease, in children with the early juvenile form, with early clinical manifestations of the disease, who still ย have the ability to walk independently and before the onset of cognitive decline.
โ ๏ธ Patient safety
No
Official EMA page
https://www.ema.europa.eu/en/medicines/human/EPAR/libmeldy