Agamree (vamorolone) - Santhera
Duvyzat (givinostat) - Italfarmaco
Adrenal insufficiency in individuals with Duchenne muscular dystrophy treated with glucocorticoids: Insights from the past, current challenges, and future directions. (PubMed, J Neuromuscul Dis) - Sep 23, 2026 - "Despite a rapidly evolving therapeutic landscape that now includes multiple disease modifying treatments including exon skipping medications, microdystrophin gene therapy, and givinostat, high dose glucocorticoids (GCs) initiated at a young age remain central to the standard of care for DMD...The approach described in this document also applies to individuals with related dystrophinopathies including Becker muscular dystrophy and female manifesting carriers treated with GCs, recognizing that GCs are not standard of care for these conditions and are prescribed on an individualized basis at some centers to severely affected individuals. Key themes discussed include best practices for individual and caregiver education about adrenal suppression, creation and implementation of stress steroid plans, safe transition between GC treatment regimens, and discontinuation of GC therapy." 
Journal • Review • Becker Muscular Dystrophy • Duchenne Muscular Dystrophy • Endocrine Disorders • Gene Therapies • Genetic Disorders • Muscular Dystrophy • Nephrology • Renal Disease • Respiratory Diseases
http://www.ncbi.nlm.nih.gov/pubmed/42770351
 
Alberto Ferlin; Alexandra Ahmet; Anne Marie Sbrocchi; Aravindhan Veerapandiyan; Carol Lam; Chiara Panicucci; Claire Wood; Cuixia Tian; Danilo Fintini; David R Weber; Eugenio Maria Mercuri; Francesca Cumbo; Gianluca Tornese; Hugh J McMillan; Jaclyn Tamaroff; Janet L Crane; Julia Sorbara; Kathi Kinnett; Kathryn A Selby; Kim Phung; Laura McAdam; Leanne M Ward; Luca Bello; Luisa De Sanctis; Maria-Elena Lautatzis; Meilan M Rutter; Melissa Fiscaletti; Mena Scavina; Nadia Merchant; Nat Nasomyont; Natascia Di Iorgi; Nora Renthal; Paula R Clemens; Rachel Schrader; Robert W Benjamin; Sabrina Corbetta; Shipra Bansal; Stefan Nicolau; Susan Apkon; Sze Choong Wong; Tommaso Aversa
 
Sep 23, 2026
 
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