The purpose of LE051 study is to evaluate the safety, tolerability, and efficacy of LE051 intravenous therapy in DMD patients treated with exon 51 skipping therapy.
Inclusion Criteria of LE051 Exon 51 Skipping Therapy
Subjects are admitted to the study only if they meet all of the following criteria:
1. Male, 4-8 years old at the beginning of screening (including boundary values);
2. DMD subjects with a clinical diagnosis of DMD referred to the Duchenne Clinical Practice Guidelines for Progressive Muscular Dystrophy (2020 edition) and whose genetic test results were confirmed to be applicable to exon skipping at No. 51 (exon skipping at 45-50, 47-50, 48-50, 49-50, 52-58, 52-63, 50, 52 exons deletion); Subjects have evidence of a DMD phenotype, characterized by typical clinical symptoms or signs such as proximal muscle weakness, staggering gait, Gowers sign, and elevated serum creatine kinase (CK);
3. The subject and/or legal guardian are able to understand and willing to comply with the requirements and procedures of the study protocol, voluntarily participate in and sign the informed consent;
4. Systemic glucocorticoids have been used for at least 3 months prior to the start of study therapy, and there has been no significant change in the therapeutic dose or administration regimen for at least 3 months prior to the start of study therapy, and it is reasonable to predict that there will be no significant change in the therapeutic dose and administration regimen throughout the study period;
5. 6MWD >=150 m during the screening period;
6. Can tolerate muscle biopsy under anesthesia, no contraindications to biopsy. [Read More]
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