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Prednisolone therapy in Fukuyama congenital muscular dystrophy with homozygous 3 kb FKTN insertion
jRCT2031230210Active, not recruiting
- Sponsor
- Japan Agency for Medical Research and Development
- Start
- Jul 2023
- Modality
- Orthosteric Ligands → Classical Binding Small Molecules → Small Molecules
- Countries
- 1
01
Overview
Open-label, single-arm trial of prednisolone in Fukuyama congenital muscular dystrophy (FCMD), sponsored by Japan Agency for Medical Research and Development. Primary endpoint: Gross Motor Function Measure. Started Jul 2023; status: active, not recruiting.
02
Drugs
prednisolone
03
Conditions
Fukuyama congenital muscular dystrophy (FCMD)
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