Konu "Risdiplam" için Araştırma Çıktıları | TR-Dizin | WoS | Scopus | PubMed listeleme
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Evaluation of the therapeutic efficacy and tolerability of current drug treatments on the clinical outcomes of paediatric spinal muscular atrophy type 1: A systematic review
(Elsevier Science Ltd, 2023)Spinal muscular atrophy (SMA) is a severe hereditary lower motor neuron disorder characterised by degeneration of alpha motor neurons in the spinal cord, resulting in progressive weakness and paralysis of proximal muscles. ...