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1 trial matches your criteria

Pelizaeus-Merzbacher disease

Recruiting

Study on the Safety and Effects of ION356 for Patients with Pelizaeus-Merzbacher Disease

InvestigationalNo placebo
Countries:FranceFrance
  • Participants:0–17 years
  • Substances:2'-O-(2-Methoxyethyl) Modified Antisense Oligonucleotide Targeting Plp1 Pre-Mrna

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