Marked improvement in HbA1c following introduction of biosimilar insulin to treatment regimen of children and youth with type 1 diabetes in Mali: A randomised controlled trial.

Aims: Evidence on outcomes of treating type 1 diabetes (T1D) with long-acting basal insulins in low-resourced settings is lacking. This study aimed to evaluate the impact of switching children and youth with T1D in the low-income country of Mali from human insulin via syringe to long-acting biosimilar insulin glargine delivered by reusable pens combined with short-acting insulin via syringe.

Methods: A two-group parallel design randomised trial was conducted enrolling 260 youth aged <25 years, diagnosed with T1D for ≥12 months without prior use of analogue insulin.

X-Linked Hypophosphataemia and Burosumab: A Systemic Disease With a New Treatment.

X linked hypophosphataemia (XLH) is a systemic, chronic condition that significantly impairs quality of life. In XLH, a phosphate regulating endopeptidase homologue X-linked (PHEX) gene mutation leads to excess fibroblast growth factor 23 (FGF23), causing hypophosphataemia and subsequent rickets, lower limb deformity, pain and other sequelae, however there are likely other non-FGF23 mediated mechanisms contributing to disease. Burosumab is an FGF23 inhibiting monoclonal antibody that has been shown to be significantly more effective in treating X linked hypophosphataemia than previously available treatment ("conventional therapy" with oral phosphate and active vitamin D).

Dental Arch Relationship Outcomes of 5-Year-Olds Born With Unilateral Cleft Lip and Palate Following the Centralization of Cleft Services.

To investigate the interdental arch relationship outcomes of 5-year-old children with unilateral cleft lip and palate (UCLP) before and after centralization of cleft services in the United Kingdom (UK) using the modified Huddart-Bodenham index (MHBI) and to investigate any differences in MHBI by cleft laterality. Retrospective cross-sectional study. Evaluation of three-dimensional study models of children with a complete UCLP.

Family planning and preimplantation testing: family experiences in congenital adrenal hyperplasia.

Introduction: Pre-implantation testing (PGT) is often suggested by healthcare professionals (HCP) to parents of children with congenital adrenal hyperplasia (CAH) considering subsequent children. Despite this, some families choose to conceive naturally without genetic testing and intervention. The aims of this study were to explore fertility choices of couples with a child with CAH and the decision making process and perceptions behind these choices, and to explore the families' lived experiences with CAH and the couples' subsequent fertility journey.

Predicting Syndromic Status Based on Longitudinal Data from Parental Reports of the Presence of Additional Structural and Functional Anomalies in Children Born with an Orofacial Cleft.

Orofacial clefts are the most common craniofacial congenital malformation in humans. Approximately 30% of clefts arise as part of a syndrome or sequence, characterised by co-existing structural and functional anomalies. Many syndromes are thought to be undiagnosed, although the presence of multiple anomalies may indicate the presence of a syndrome or sequence.