New hope for DMD? small study tests immune drug to slow muscle decline

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: 1. Participant age: 6-10 years old (including 10 years old). 2. Gender: male. 3. Gene diagnosis of DMD gene type, conform to the DMD phenotypes and clinical manifestations. 4. There has been a muscle weakness, symptoms of motor function decline. 5. Patients could walk independently, able to complete 10 meters walk test. Lie on your back up, 6. patients can complete independently and got up time is less than 30 seconds. 7. Participants cognitive assessment and mental state can cooperate movement. 8. Never accept oral hormone therapy or stop using hormone therapy more than six months. 9. Willing to accept and be able to cooperate with more than one muscle biopsies. Exclusion Criteria: 1. The participants are in active virus infection or other pathogen infection, including but not limited to, the TORCH virus, Epstein - Barr virus, the new crown virus, bacteria, fungi, etc. 2. Having received a live attenuated vaccine or systemic antiviral, anti-infective and/or interferon therapy within 3 months prior to the treatment with investigational product. 3. Serological tests revealed infections of HIV, HBV, HCV and syphilis. 4. Severe infections (such as pneumonia, pyelonephritis or meningitis) occurred within 8 weeks before the start of treatment with investigational product. 5. There are clear symptoms of cardiomyopathy, and echocardiography shows that the left ventricular ejection fraction is less than 50%. 6. Continuous or intermittent assistance support from a ventilator is required. 7. Laboratory biochemical tests the following indices abnormal: gamma GGTP (gamma glutamyl transpeptidase) 2 times higher than upper limit (GGT), total bilirubin is higher than 1.5 times the upper limit of the elf inhibition C (cystatin C) \> 1.27 mg/L, hemoglobin (Hgb) \< 100 g/L or \> 200 g/L; White blood cell (WBC) \<4×109/L or \>18.5×109/L or platelet ≤125×109/L. 8. The patient has received any type of gene therapy (such as AAV gene therapy), cell therapy (such as stem cell transplantation), in vivo editing or in vitro editing reinfusion gene editing therapy (such as CRISPR-Cas9, TALEN), or other experimental drug treatments in the past. 9. Participants have any taboos on immunosuppressive therapy. 10. Other comorbid diseases or conditions that the principal investigators considered unsuitable for participation in clinical trials. 11. The families of the participants do not wish to publicly disclose the patients' research participation to the attending physician and other medical providers.