Registered titles

Below is a list of titles that have been registered, but not yet published as protocols.

  • Circadian hydrocortisone replacement regimens for congenital adrenal hyperplasia
  • Combined large neutral amino acid supplementation for phenylketonuria
  • Desmopressin (DDAVP) for for preventing and treating bleeding in people with mild and moderate haemophilia
  • Dietary interventions for maple syrup urine disease
  • Enzyme replacement therapy for late onset Pompe's disease
  • Enzyme replacement therapy for mucopolysaccharidosis type IV (Morquio syndrome)
  • Growth hormone therapy for people with thalassaemia
  • Hydroxyurea for beta-thalassemia major
  • Inhaled muco-active agents for cystic fibrosis: an overview of Cochrane systematic reviews
  • Interventions for chronic kidney disease in people with sickle cell disease
  • Interventions for improving adherence to iron chelation therapy in people with sickle cell disease or thalassaemia
  • Interventions for managing infertility in people with beta-thalassaemia major or intermedia
  • Interventions for promoting participation in shared decision making for children with cystic fibrosis¬†
  • Non-CFTR mediated chloride secretagogues for cystic fibrosis lung disease
  • Red blood cell transfusion to treat or prevent complications in sickle cell disease: an overview of systematic reviews
  • Sweat test for the diagnosis of cystic fibrosis in all age groups (DTA)
  • Therapies to prevent recurrence of Pseudomonas aeruginosa in people with cystic fibrosis
  • Treatment of dental complications in thalassaemia
  • Transcutaneous electrical nerve stimulation (TENS ) and biofeedback as complementary and alternative therapies for managing pain in sickle cell disease