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...

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Bibliographic Details
Main Authors: Aleksandra Alicja Majchrzak-Celińska (Author), Anna Warych (Author), Mikołaj Szoszkiewicz (Author)
Format: Book
Published: Polish Pharmaceutical Society, 2020-02-01T00:00:00Z.
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3rd Floor Main Library

Holdings details from 3rd Floor Main Library
Call Number: A1234.567
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