Pórszász, RóbertAnanyan, Edgar2024-04-192024-04-192024-02-25https://hdl.handle.net/2437/369072Duchenne muscular dystrophy (DMD) is a progressive, debilitating disorder that results from a mutation present on the X chromosome, leading to the inability to produce functional dystrophin protein. Progressive tissue damage accumulates with every muscle contraction, caused by the lack of dystrophin, and leads to loss of muscle strength, chronic inflammation, and fibrosis. Current treatment involves administration of corticosteroids and management of cardiac and respiratory symptoms as the disease progresses. However, current treatment only slows down the progression of the disease with significant side effects. Therefore multiple new drugs are undergoing trials to either slow down the symptom progression or treat the underlying cause of the disease. In this thesis, we aim to discuss the efficacy, mode of action, and tolerability of newly emerging treatments that could potentially benefit DMD patients.43enDMDTreatment of Duchenne muscular dystrophyCurrent treatment of Duchenne muscular dystrophyEmerging treatments of Duchenne muscular dystrophyMedicine::PharmacologyHozzáférhető a 2022 decemberi felsőoktatási törvénymódosítás értelmében.