Natural disease course modeling of achondroplasia to evaluate the efficacy of recifercept in the absence of a placebo control arm in phase II study
Abstract While randomized, placebo‐controlled, double‐blinded clinical studies are the gold standard for evaluating the efficacy of investigational drugs, the use of placebo in children with achondroplasia should be limited because it provides no clinical benefit while exhausting study participants&...
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| Main Authors: | , , , |
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| Format: | Book |
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Wiley,
2024-07-01T00:00:00Z.
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| Online Access: | Connect to this object online. |
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