SMA Therapy in Poland: New Hopes and Challenges

• Main Authors: Iwona Welian (Author), Karolina Gendek (Author), Ilona Jastrzębska (Author), Karolina Maliszewska (Author), Magdalena Chrościńska-Krawczyk (Author)
• Format: Book
• Published: Kazimierz Wielki University, 2023-04-01T00:00:00Z.
• Subjects: article
• Online Access: 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...