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What are the effects of blood transfusion for preventing stroke in children and adolescents with sickle cell disease?
How do oral non-steroidal anti-inflammatory drugs affect outcomes in people with cystic fibrosis lung disease?
How does dornase alfa compare with placebo in people with cystic fibrosis?
In adults with Charcot-Marie-Tooth IA disease, is there randomized controlled trial evidence to support the use of ascorbic acid?
In children with cystic fibrosis, how does bronchoscopy-guided antimicrobial therapy compare with standard antimicrobial therapy at improving outcomes?
What are the effects of drug interventions for preventing the progression of autosomal dominant polycystic kidney disease in adults?
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