Spinal muscular atrophy - onasemnogene abeparvovec and other therapeutic options

• Huvudskapare: Aleksandra Alicja Majchrzak-Celińska (Författare, medförfattare), Anna Warych (Författare, medförfattare), Mikołaj Szoszkiewicz (Författare, medförfattare)
• Materialtyp: Bok
• Publicerad: Polish Pharmaceutical Society, 2020-02-01T00:00:00Z.
• Ämnen: article
• Länkar: Connect to this object online.

Spinal muscular atrophy (SMA) is a neuromuscular disorder that results in the loss of motor neurons. SMA is caused by mutations in the SMN1 gene, leading to the decreased synthesis of the SMN protein, necessary for motor neuron survival. In the past, SMA was considered to be an incurable disease, an...