Spinal muscular atrophy - onasemnogene abeparvovec and other therapeutic options
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...
| Main Authors: | , , |
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| Format: | Article |
| Language: | Polish |
| Published: |
Polish Pharmaceutical Society
2020-02-01
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| Series: | Farmacja Polska |
| Subjects: | |
| Online Access: | https://www.ptfarm.pl/download/?file=File%2FFarmacja+Polska%2F2020%2F1%2F02_OG_Rdzeniowy_zanik_miesni_n.pdf |