The social-economic burden of spinal muscular atrophy in Russia

• Egile Nagusiak: A. S. Kolbin (Egilea), D. V. Vlodavets (Egilea), A. A. Kurylev (Egilea), Yu. Ye. Balykina (Egilea), M. A. Proskurin (Egilea), S. A. Mishinova (Egilea), O. Yu. Germanenko (Egilea), N. Yu. Kolbina (Egilea)
• Formatua: Liburua
• Argitaratua: IRBIS LLC, 2021-02-01T00:00:00Z.
• Gaiak: article
• Sarrera elektronikoa: Connect to this object online.

Introduction. Spinal muscular atrophies (SMA) are clinically and genetically heterogeneous congenital orphan diseases that lead to progressive spinal motoneurons degeneration and loss of their function. There are 4 types of SMA with type I being the most severe. SMA patients need lots of services (m...