Core Insights - Duchenne Muscular Dystrophy (DMD) affects approximately 70,000 patients in China, with a prevalence of 1 in every 5,000 newborn boys [1][2] - The standard treatment with corticosteroids can extend median survival by over 10 years, yet only 23% of patients in China receive this treatment [1][6] - The approval of Vamorolone, the first innovative corticosteroid for DMD in China, marks a significant advancement in treatment options [1][7] Patient Statistics - DMD is a genetic disorder caused by mutations in the DMD gene, leading to progressive muscle degeneration and associated complications [2] - Globally, there are about 250,000 DMD patients, with China having one of the highest numbers [2] - Approximately 30% of DMD cases are due to gene mutations, while around 70% of affected children have carrier mothers [2] Treatment Landscape - The current corticosteroid treatment rate in China has increased from 26.3% in 2015 to 54% in 2020, but nearly half of the patients have never used medication [6] - The lack of treatment is partly due to the absence of approved drugs, with Deflazacort only available through temporary importation [6][7] - Vamorolone, approved in December 2022, is expected to participate in national health insurance negotiations after passing the basic directory review [7] Gene Therapy Developments - Gene therapy is emerging as a significant area in DMD drug development, with various drugs approved globally, but only one gene therapy product has been approved [8][9] - Elevidys, a gene therapy drug, was approved by the FDA for DMD patients aged 4-5 years, but its high cost and associated controversies have raised concerns [9] - BBM-D101, a domestic gene therapy candidate, has received IND approval and is currently in clinical trials [9]