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Measuring the effect of newborn screening on survival after haematopoietic cell transplantation for severe combined immunodeficiency: a 36-year longitudinal study from the Primary Immune Deficiency Treatment Consortium.
Safety and efficacy of mitapivat, an oral pyruvate kinase activator, in adults with non-transfusion dependent a-thalassaemia or �-thalassaemia: an open-label, multicentre, phase 2 study.
Mini-outbreak response for malaria using indoor residual spraying.
Once-daily, subcutaneous vosoritide therapy in children with achondroplasia: a randomised, double-blind, phase 3, placebo-controlled, multicentre trial.
Transparency and diversity in cystic fibrosis research.
Effectiveness of reactive focal mass drug administration and reactive focal vector control to reduce malaria transmission in the low malaria-endemic setting of Namibia: a cluster-randomised controlled, open-label, two-by-two factorial design trial.
Are three drugs for malaria better than two?
A shorter course for anti-relapse therapy against vivax malaria.
Gender norms and health: insights from global survey data.
Burosumab versus conventional therapy in children with X-linked hypophosphataemia: a randomised, active-controlled, open-label, phase 3 trial.