Orchard Therapeutics (Netherlands) B.V.
SpainAll drugs from this manufacturer
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It is used to treat metachromatic leukodystrophy (MLD) in children with the 'late infantile' or 'early juvenile' forms who are currently asymptomatic, or in children with the 'early juvenile' form who have already begun to show symptoms but without rapid progression.
Dosage form: solution for injection
Active substance Dosage atidarsagen autotemcel 2-10 million células/ml