Evaluation of the Safety and Efficacy of BBM-D101 to Treat Patients with Duchenne Muscular Dystrophy

Prior treatment

• Receiving antiviral therapy for hepatitis B, hepatitis C, HIV, etc.;
• The subject\'s preventive treatment/cardiomyopathy treatment changes within 1 month before the start of the study treatment;

Other illness

• Hepatitis B surface antigen (HBsAg) positive, hepatitis B virus deoxyribonucleic acid (HBV-DNA) ≥1000U/mL, hepatitis C virus ribonucleic acid (HCV-RNA) positive or human immunodeficiency virus (HIV) positive;
• With severe or persistent arrhythmias and congenital heart disease.

Lab values

• Left ventricular ejection fraction (LVEF) \<50% or ≥ class III cardiac function defined by New York Heart Association (NYHA);
• With underlying liver disease, such as previous diagnosis of portal hypertension, splenomegaly, hepatic encephalopathy, or hepatic fibrosis ≥ stage 3; or nodules, cysts found by B-ultrasound in the past, or elevated alpha-fetoprotein in laboratory tests during the screening period, etc., and these abnormalities are judged by the investigator to be clinically significant;

• The legal guardian of the subject fully understands the purpose, nature, methods, and possible risks of the study, and signs a written informed consent form;
• The study includes ambulatory male subjects who are at least 4 years old and less than 8 years old (4 years old ≤ age \< 8 years old) ;
• Genetically confirmed diagnosis of DMD;
• Have at least 1 of the following typical clinical signs or laboratory abnormalities of DMD: proximal muscle weakness, waddling gait, pseudo gastrocnemius hypertrophy, Gower\'s sign, pterygoid scapula;
• Ability to cooperate with motor assessment testing, magnetic resonance imaging (MRI) and muscle biopsy according to the requirements of the study.