Core Insights - Santhera Pharmaceuticals presented long-term real-world data on AGAMREE® (vamorolone) at the MDA Clinical & Scientific Conference 2026, demonstrating its effectiveness and improved safety profile compared to traditional corticosteroids for treating Duchenne muscular dystrophy (DMD) [1][3][12]. Long-Term Effectiveness - Vamorolone shows comparable long-term effectiveness to classic corticosteroids, with no statistically significant difference in time to loss of ambulation between vamorolone and traditional treatments [5][9]. - The mean real-world dose of vamorolone was 4.5 ± 1.8 mg/kg/day, with a median exposure of approximately five years, indicating that patients may tolerate higher doses over the long term [5]. Growth and Development - Patients treated with vamorolone maintained height trajectories, achieving a mean height advantage of +12.17 cm compared to those on classic corticosteroids, where growth stunting was observed [6][9]. - Vamorolone is the first dissociative corticosteroid to demonstrate that patients with DMD can achieve normal growth without compromising efficacy [6]. Safety Profile - There was an 80% reduction in the proportion of patients with vertebral fractures in the vamorolone group (8.1%) compared to those treated with deflazacort (41.9%) [7][9]. - Non-vertebral fractures occurred in 27.5% of patients, indicating a favorable profile for chronic use compared to traditional corticosteroids [8]. - Cataracts were observed in significantly fewer patients treated with vamorolone (5.3%) compared to those on deflazacort (37.8%) [10][9]. Ongoing Research - The GUARDIAN study will continue to assess long-term outcomes, including muscle function, bone health, and additional safety endpoints, contributing to the understanding of vamorolone's long-term benefit-risk profile [11][12]. Company Overview - Santhera Pharmaceuticals focuses on developing innovative medicines for rare neuromuscular diseases, with AGAMREE® positioned as a dissociative anti-inflammatory drug and an alternative to classic corticosteroids for DMD [17].