SMA Therapy in Poland: New Hopes and Challenges

• 主要な著者: Iwona Welian (著者), Karolina Gendek (著者), Ilona Jastrzębska (著者), Karolina Maliszewska (著者), Magdalena Chrościńska-Krawczyk (著者)
• フォーマット: 図書
• 出版事項: Kazimierz Wielki University, 2023-04-01T00:00:00Z.
• 主題: article
• オンライン･アクセス: Connect to this object online.

Abstract: Spinal muscular atrophy (SMA) is a genetically inherited disease whose symptoms appear in children at a fairly early age. The main cause of the disease is a mutation of the SMN1 gene, which causes the lack of production of SMN This results in the disappearance of motoneurons, which consequ...