Genetic Modifier Associations With Steroid Safety Outcomes in Duchenne Muscular Dystrophy (ICNMD 2026) - Jul 6, 2026 - Abstract #PP01.42; Pres time: Jul 8, 2026; 10:30 AM - 11:15 AM; Location: Spadolini - Ground Floor; "Traditional corticosteroids like prednisone and deflazacort can result in weight gain, adrenal suppression, growth stunting, and reduced bone biomarkers, while vamorolone, a newly approved dissociative steroid, has shown an improved safety profile, especially on bone health. Al though based on a small sample size and needing additional studies for confirmation, we provide initial evidence of the association of genetic modifiers at an early age with common steroid safety concerns including for weight gain, which is a common reason for steroids prescribed at sub-recommended doses. These findings may be important for clinical trials and care."
Clinical • CNS Disorders • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy • CDX2
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Ms. Fatemeh Ahmadiharchegani; Prof. Eric Hoffman; Dr. Daniele Sabbatini; Prof. Elena Pegoraro; Prof. Luca Bello; Prof. Michela Guglieri; Dr. Paula Clemens; Prof. Utkarsh Dang
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