Duchenne Muscular Dystrophy
Conditions
Interventions
patients with Duchenne muscular dystrophy:NA
Sponsors
Peking University First Hospital
Eligibility
Sex/Gender
Male
Age
No minimum to 18 Years
Inclusion criteria
Inclusion criteria: 1. Chinese male subjects diagnosed with DMD. The diagnosis conclusion of the subject must be recorded in his medical history, and must be confirmed by the Genetic testing results reported by the corresponding supervised laboratory using clinically verified Genetic testing methods for the purpose of disease diagnosis during routine clinical diagnosis and treatment. The results of Genetic testing must be able to confirm the existence of mutations in the dystrophin gene and be consistent with the diagnosis conclusion of DMD. 2. A signed and dated informed consent form and consent form (if applicable), which is used as evidence to show that the subject and his legal representative/parents/Legal guardian have been informed of all research related information. 3. Subjects and their Legal guardian are willing and able to participate in the planned laboratory examination and other research steps as required. Subjects agreed to participate in this study according to local regulations and the requirements of the Institutional review board (IRB).
Exclusion criteria
Exclusion criteria: 1. Other systemic Immunosuppressive drug other than glucocorticoids are being used. 2. The subject has previously used any gene therapy medication. 3. Family members of research center staff directly involved in the implementation of this study who are managed by the researcher, as well as research center staff who are not managed by the researcher, or family members of Pfizer employees directly involved in the implementation of this study.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The incidence of AAV9 NAb positivity in Chinese DMD subjects; | — |
Secondary
| Measure | Time frame |
|---|---|
| The frequency of aav9 nba positive and different titers in various demographic subgroups; | — |
Countries
China
Contacts
Public ContactYuwu Jiang
Peking University First Hospital
Outcome results
None listed