autologous cd34+ cells encoding the arsa gene

Italy

Drugs containing this active substance

  • LIBMELDY

    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.

    Form: solution for infusion

    Active substance Dosage
    autologous cd34+ cells encoding the arsa gene -

    ATC: A16AB21

    Manufacturer: ORCHARD THERAPEUTICS (NETHERLANDS) B.V.