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New Clinical Genetics research.

The strongest recent Clinical Genetics papers on PubMed, ranked by study design, topic and journal, with the same summaries subscribers get.

  1. randomized trial, 207 patients

    Efficacy and safety of mitapivat in patients with sickle cell disease (RISE UP): a phase 3, double-blind, global, randomised, placebo-controlled trial

    Andemariam B, Kuo KHM, Algahtani FH, et al. Lancet. Oct 2026.

    Findings
    In the trial, 56 (41%) of 138 mitapivat‑treated patients achieved a ≥1 g/dL hemoglobin increase versus 2 (3%) of 69 on placebo, and the annualised pain crisis rate was 2·6 versus 3·1 per year.
    Bottom line
    Mitapivat yields a 38% higher hemoglobin response rate (56/138 vs 2/69) without significantly reducing pain crises.

Summaries are written by AI from PubMed abstracts, for keeping up with the literature. Check the source before it changes care. Editorial policy · Medical disclaimer

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