Duchenne Muscular Dystrophy Clinical Trial
Official title:
Efficacy of Umbilical Cord Mesenchymal Stem Cells in Duchenne Muscular Dystrophy: Phase 1/2 Study
Duchenne muscular dystrophy (DMD) is a genetic disorder caused by an absence of dystrophin
and characterized by progressive muscle degeneration. There is no cure for DMD at present
but, there are several strategies under-researched for treatment of DMD such as steroid
treatment, gene theraphy, exon skipping, stop codon read through and gene repair, cell
theraphy and theraphy with drug that help to produce utrophin protein.
The aim of this study is investigate the eficacy of human umblical cord mesenchymal stem
cells on DMD and understanding if wild type gene can be transfered to the patient.
n/a
Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
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