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Deflazacort treatment in LMNA-related congenital muscular dystrophy
2019-004426-24Phase 2Active
- Sponsor
- Azienda Ospedaliero-Universitaria Pisana
- Start
- Jun 2021
- Modality
- Orthosteric Ligands → Classical Binding Small Molecules → Small Molecules
- Countries
- 1
01
Overview
Phase 2, open-label, single-arm trial of deflazacort in congenital muscular dystrophy (CMD), Emery-Dreifuss muscular dystrophy type 2 (EDMD2) and muscle atrophy, sponsored by Azienda Ospedaliero-Universitaria Pisana. Primary endpoint: Safety evaluation of deflazacort oral drops. Started Jun 2021; status: active.
02
Drugs
deflazacort
03
Conditions
congenital muscular dystrophy (CMD)Emery-Dreifuss muscular dystrophy type 2 (EDMD2)muscle atrophy
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