Health economic evaluation of risdiplam in patients with spinal muscular atrophy

Background. Spinal muscular atrophies (SMA) are clinically and genetically heterogenous inherited orphan diseases leading to progressive spinal motoneurons degeneration and loss of function. Risdiplam and nusinersen are both authorized in Russia for pathogenic SMA treatment and included in the list...

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Main Authors: А. S. Kolbin (Author), А. А. Kurylev (Author), Yu. Е. Balykina (Author), М. А. Proskurin (Author), S. А. Mishinova (Author)
Format: Book
Published: IRBIS LLC, 2021-10-01T00:00:00Z.
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3rd Floor Main Library

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Call Number: A1234.567
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