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Deferiprone in Pelizaeus-Merzbacher disease (proof-of-concept)
2024-511968-81-00Phase 2Authorised
- Sponsor
- Amsterdam University Medical Center
- Start
- Dec 2024
- Modality
- Small Molecules
- Countries
- 1
01
Overview
Phase 2, single-arm trial of deferiprone in Pelizaeus-Merzbacher disease (PMD), sponsored by Amsterdam University Medical Center. Primary endpoint: Motor function (GMFM-88 and MLD-GMF score). Planned start Dec 2024; status: authorised.
02
Drugs
deferiprone
03
Conditions
Pelizaeus-Merzbacher disease (PMD)
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