Metachromatic Leukodystrophy

• EMA approval · 2025-12-16
  EMA Authorised: Libmeldy (atidarsagene autotemcel) — Orchard Therapeutics (Netherlands) B.V.

  The European Medicines Agency authorised Libmeldy (atidarsagene autotemcel) from Orchard Therapeutics (Netherlands) B.V. for metachromatic l…

• EMA approval · 2020-10-16
  New gene therapy to treat rare genetic disorder metachromatic leukodystrophy

  EMA announces a new gene therapy to treat metachromatic leukodystrophy, a rare genetic disorder, marking a significant treatment option adva…