Long-term clinical outcomes with Libmeldy in children with early-onset MLD
Gene therapy developer Orchard Therapeutics (Nasdaq: ORTX) has announced the publication in The Lancet of long-term clinical outcomes evaluating the safety and efficacy of Libmeldy (atidarsagene autotemcel) for the treatment of early-onset metachromatic leukodystrophy (MLD).
Libmeldy is the only approved one-time gene therapy intended to correct the underlying cause of MLD for eligible patients in the European Union, UK, Iceland, Liechtenstein and Norway. Also known as OTL-200, it is an investigational therapy in the USA.
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