Duchenne Muscular Dystrophy
Conditions
Keywords
umbilical cord, mesenchymal stem cell
Brief summary
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.
Interventions
BIOLOGICALUmbilical Cord Mesenchymal Stem Cell
Sponsors
Acibadem University
Study design
Allocation
RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
MALE
Age
7 Years to 20 Years
Healthy volunteers
No
Inclusion criteria
\- Patients with diagnosis of DMD that is proven clinically and genetically Age between 7-20 Patients need partial respiratory support, during the day Patients have less than or equal to stage I NIH, Liver, renal and cardiac function Patients without cancer Patients without allergic disease Patients without bleeding diathesis,
Exclusion criteria
Patients need complete respiratory support Patients have more than to stage II NIH, Liver, renal and cardiac function Patients have bleeding diathesis and allergic disease
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Duchenne muscular dystrophy gene expression | up to 9 months |
Countries
Turkey (Türkiye)
Contacts
Primary ContactErcument Ovali, Prof.Dr.
Backup ContactCengiz Yakicier, Prof.Dr.
Outcome results
None listed