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Libmeldy
autologous CD34+ cells encoding ARSA gene
Leucodistrofia metacromatica
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Autorizzato
Terapia avanzata
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Nessun allarme attivo
Questo medicinale non presenta carenze in corso, lettere di sicurezza o richiami.
A cosa serve
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 Sicurezza del paziente
No
Pagina ufficiale EMA
https://www.ema.europa.eu/en/medicines/human/EPAR/libmeldy