Efficacy and Safety of Vamorolone Over 48 Weeks in Boys With Duchenne Muscular Dystrophy: A Randomized Controlled Trial.
Utkarsh J DangJesse M DamskerMichela GuglieriPaula R ClemensSeth J PerlmanEdward C SmithIain HorrocksRichard S FinkelJean K MahNicolas DeconinckNathalie M GoemansJana HaberlováVolker StraubLaurel Mengle-GawBenjamin D SchwartzAmy HarperPerry B ShiehLiesbeth De WaeleDiana CastroMichele L YangMonique M RyanCraig M McDonaldMar TuliniusRichard I WebsterHugh J McmillanNancy KuntzVamshi K RaoGiovanni BaranelloStefan SpintyAnne-Marie ChildsAnnie M SbrocchiKathryn A SelbyMigvis MonduyYoram NevoJuan Jesus VilchezAndres Nascimento-OsorioErik H NiksImelda J M De GrootMarina KatsalouliJohn N Van Den AnkerLeanne M WardMika LeinonenAndrea L D'AlessandroEric P HoffmanPublished in: Neurology (2024)
This study provides Class I evidence that for boys with DMD, the efficacy of vamorolone at a dose of 6 mg/kg/d was maintained over 48 weeks.