ORCHARD THERAPEUTICS (NETHERLANDS) B.V.
ItalyAll drugs from this manufacturer
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Libmeldy is a gene therapy used to treat metachromatic leukodystrophy (MLD) in children with 'late infantile' or 'early juvenile' forms who have not yet shown symptoms, or in children with the 'early juvenile' form who have begun to show symptoms but are not deteriorating rapidly.
Dosage form: solution for infusion
Active substance Dosage autologous cd34+ cells encoding the arsa gene - ATC: A16AB21