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Expanded access study of OTL-200 in patients with early-onset Metachromatic-leukodystrophy

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Trial Profile

Expanded access study of OTL-200 in patients with early-onset Metachromatic-leukodystrophy

Status: Completed
Phase of Trial: Clinical Phase Unknown

Latest Information Update: 19 Mar 2024

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At a glance

Most Recent Events

  • 18 Mar 2024 The U.S. Food and Drug Administration approved Lenmeldy (atidarsagene autotemcel) to Orchard Therapeutics, the first FDA-approved gene therapy indicated for the treatment of children with pre-symptomatic late infantile, pre-symptomatic early juvenile or early symptomatic early juvenile metachromatic leukodystrophy (MLD).
  • 18 Mar 2024 Results published in the Media Release
  • 24 Feb 2023 According to an Orchard Therapeutics media release, data of this trial used for Long-term integrated analysis are being presented at the ongoing 19th Annual WORLDSymposium™ taking place February 22-26, 2023, in Orlando, Florida.

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