Core Viewpoint - The article emphasizes the importance of awareness and management of Duchenne Muscular Dystrophy (DMD), highlighting the need for improved treatment options and support for affected families [1][2][4]. Group 1: Disease Overview - DMD is a severe muscle degeneration disease caused by X-linked recessive inheritance, primarily affecting males, with an estimated prevalence of 1 in 5,000 newborn boys globally [2]. - There are approximately 250,000 DMD patients worldwide, with around 70,000 in China, making it one of the countries with the highest number of DMD patients [2]. Group 2: Disease Progression and Management - DMD progresses through five stages based on age and mobility, with a median age of death in China at approximately 18.57 years, while current standard treatments extend median survival to 28.7 years, an increase of over 10 years compared to untreated patients [3]. - Multidisciplinary management can delay disease progression and improve quality of life, with standardized treatment showing survival rates between 24.8 and 27.9 years [3]. Group 3: Treatment Challenges - The standard treatment adherence rate for DMD patients is only 23%, with a lack of specialized medical resources leading to inadequate treatment and management [7]. - Long-term use of traditional corticosteroids can result in adverse effects, prompting a demand for better, less harmful treatment options [7]. Group 4: New Developments - The approval of the first DMD-specific drug, Vamorolone, in December 2024 marks a significant advancement in treatment options, transitioning patients from having no available treatments to a new era of standardized care [7]. - Over 40 clinical research projects for DMD treatments are currently underway globally, indicating a promising future for patient longevity [7]. Group 5: Financial and Policy Concerns - High drug prices and insufficient insurance coverage pose significant barriers for DMD families, with over 70% of families spending more than 60% of their income on medical expenses [8]. - Calls for improved insurance coverage and legislative support for rare diseases are emphasized to ensure better access to treatment for patients [8]. Group 6: Community Engagement - The DMD Annual Summit and empowerment training aim to raise awareness and provide resources for patients and families, encouraging community involvement in DMD advocacy [9].

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]

Core Insights - The treatment field for Duchenne Muscular Dystrophy (DMD) has made progress with the clinical application of the innovative drug Vamorolone at Beijing Children's Hospital, marking the first prescription issued in China since its approval [1][2] Group 1: Drug Development and Approval - Vamorolone was approved by the National Medical Products Administration in December 2024 for the treatment of DMD in patients aged four and above [2] - The drug is noted for its anti-inflammatory properties and has fewer side effects compared to traditional corticosteroids, which are commonly used in DMD treatment [2] Group 2: Patient Impact and Treatment Challenges - A 9-year-old patient, diagnosed with DMD five years ago, has been using corticosteroids and rehabilitation to manage the disease progression [1] - Long-term use of corticosteroids can lead to side effects such as osteoporosis and stunted growth, which Vamorolone aims to mitigate [2] Group 3: Disease Overview - DMD is an X-linked recessive genetic disorder primarily affecting boys, with an incidence of approximately 1 in every 3,500 newborn males globally [1] - The disease is characterized by progressive muscle weakness, with symptoms typically becoming apparent by age three, leading to loss of independent mobility by ages 10-12 [1]