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Libmeldy
autologous CD34+ cells encoding ARSA gene
Leucodistrofia metacromática
✅ Autorizado
Terapia avançada
✅
Nenhum alerta ativo
Este medicamento não apresenta escassez contínua, cartas de segurança ou recolhimentos.
Para que 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 have the ability to walk independently and before the onset of cognitive decline.
⚠️ Segurança do paciente
No
Página oficial da EMA
https://www.ema.europa.eu/en/medicines/human/EPAR/libmeldy