How does risdiplam compare with other treatments for Types 1–3 spinal muscular atrophy: a systematic literature review and indirect treatment comparison
To conduct indirect treatment comparisons between risdiplam and other approved treatments for spinal muscular atrophy (SMA). Individual patient data from risdiplam trials were compared with aggregated data from published studies of nusinersen and onasemnogene abeparvovec, accounting for heterogeneit...
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Published in | Journal of comparative effectiveness research Vol. 11; no. 5; pp. 347 - 370 |
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Main Authors | , , , , , , , , |
Format | Journal Article |
Language | English |
Published |
England
Future Medicine Ltd
01.04.2022
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Subjects | |
Online Access | Get full text |
ISSN | 2042-6305 2042-6313 2042-6313 |
DOI | 10.2217/cer-2021-0216 |
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Summary: | To conduct indirect treatment comparisons between risdiplam and other approved treatments for spinal muscular atrophy (SMA).
Individual patient data from risdiplam trials were compared with aggregated data from published studies of nusinersen and onasemnogene abeparvovec, accounting for heterogeneity across studies.
In Type 1 SMA, studies of risdiplam and nusinersen included similar populations. Indirect comparison results found improved survival and motor function with risdiplam versus nusinersen. Comparison with onasemnogene abeparvovec in Type 1 SMA and with nusinersen in Types 2/3 SMA was challenging due to substantial differences in study populations; no concrete conclusions could be drawn from the indirect comparison analyses.
Indirect comparisons support risdiplam as a superior alternative to nusinersen in Type 1 SMA.
How does risdiplam compare with other treatments for Types 1–3 spinal muscular atrophy? A systematic literature review and indirect treatment comparison. |
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Bibliography: | ObjectType-Article-1 SourceType-Scholarly Journals-1 ObjectType-Feature-2 ObjectType-Review-3 content type line 23 ObjectType-Undefined-4 |
ISSN: | 2042-6305 2042-6313 2042-6313 |
DOI: | 10.2217/cer-2021-0216 |