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4. Safety and efficacy of tamoxifen in boys with Duchenne muscular dystrophy (TAMDMD): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial

8. Retrospective, Landmark Analysis of Long-term Adult Morbidity Following Allogeneic HSCT for Inborn Errors of Immunity in Infancy and Childhood

11. Relationship between growth and ambulation loss in Duchenne muscular dystrophy boys on steroids.

12. Influence of Sex, Race and Ethnicity, and Deprivation on Survival and Completion of the Fontan Pathway for Children With Functionally Single Ventricle Heart Disease.

16. mRNA therapy corrects defective glutathione metabolism and restores ureagenesis in preclinical argininosuccinic aciduria

18. Quantifying additional procedures in functionally single ventricle disease: a national cohort study

20. Risk Factors for Reintervention With Functionally Single-Ventricle Disease Undergoing Staged Palliation in England and Wales: A Retrospective Cohort Study

21. Safety and efficacy of tamoxifen in boys with Duchenne muscular dystrophy (TAMDMD): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial

26. Surgical Management of Submucous Cleft Palate by Radical Muscle Dissection Veloplasty: Speech Outcomes in Patients with 22q11.2 Deletion Syndrome.

29. Long-term Natural History of Pediatric Dominant and Recessive RYR1 Related Myopathy

30. Respiratory Trajectories in Type 2 and 3 Spinal Muscular Atrophy in the iSMAC Cohort Study

34. Long-term survival and center volume for functionally single-ventricle congenital heart disease in England and Wales

35. Expanding the phenotype in argininosuccinic aciduria: need for new therapies

40. Risk Factors for Reintervention With Functionally Single-Ventricle Disease Undergoing Staged Palliation in England and Wales: A Retrospective Cohort Study.

42. PO195 Outcome measures for duchenne muscular dystrophy: implications for trials

44. Determining minimal clinically important differences in the North Star Ambulatory Assessment (NSAA) for patients with Duchenne muscular dystrophy.

45. Argininosuccinic aciduria fosters neuronal nitrosative stress reversed by Asl gene transfer

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