News & Updates

Using small molecule drugs to modulate the cystic fibrosis transmembrane conductance regulator (CFTR) protein improves multiple aspects of cystic fibrosis in paediatric patients, including lung function, CFTR protein function, and nutritional status, according to a recent meta-analysis.

Treatment with intravenous ganaxolone induces rapid and durable seizure control in patients with refractory status epilepticus, while showing acceptable safety and tolerability, according to the results of an open-label phase II trial.

In the treatment of children with obstructive sleep apnoea syndrome (OSAS), use of intranasal corticosteroids falls short of producing substantial changes in symptoms as well as in polysomnographic and neurobehavioural parameters at 3 and 12 months, a study has found.

Patients with transfusion-dependent β-thalassemia (TDT) continued to benefit from over 3 years of treatment with luspatercept, a first-in-class erythroid maturation agent that enhances late-stage erythropoiesis, according to the longer-term efficacy analysis of the phase III BELIEVE study.

High-dose zinc supplementation was significantly associated with decreased feeding intolerance in very low birth weight (VLBW) preterm infants, according to a study presented at ESPGHAN 2022.

The final 2-year (part B) results of the phase III CARDINAL study continued to demonstrate the favourable risk-benefit profile of sutimlimab, a first-in-class selective anti-C1s classical complement pathway (CP) inhibitor, for the management of cold agglutinin disease (CAD), a rare chronic, autoimmune haemolytic anaemia characterized by classical CP-mediated haemolysis.