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Rare disease

Pelizaeus-Merzbacher disease

  • 2 clinical trials
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2 Clinical trials

Countries:The NetherlandsThe Netherlands
  • Participants:0–17 years
  • Substances:Deferiprone
  • Sponsor:Amsterdam UMC Stichting
Countries:FranceFrance
  • Participants:0–17 years
  • Substances:2'-O-(2-Methoxyethyl) Modified Antisense Oligonucleotide Targeting Plp1 Pre-Mrna
  • Sponsor:Ionis Pharmaceuticals Inc.
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