新药获批，全国首张伐莫洛龙处方在京开出

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]