Pediatrics
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Oral Iptacopan Monotherapy in Paroxysmal Nocturnal Haemoglobinuria: 48-Week Outcomes from APPLY-PNH and APPOINT-PNH Phase 3 Trials
Oral iptacopan provides sustained haemolysis control and improved haemoglobin in PNH patients over 48 weeks, with a favorable safety profile, supporting its role as a new therapeutic option.
Impact of Maternal Pertussis Vaccination on Infant Immune Responses: Insights from the GaPS Trial in The Gambia
A phase 4 trial in The Gambia finds that maternal Tdap-IPV vaccination is safe and provides early infant protection, but modestly blunts immune responses to whole-cell pertussis vaccines without compromising overall immunogenicity.
Prademagene Zamikeracel: A Breakthrough Gene Therapy for Chronic Wounds in Recessive Dystrophic Epidermolysis Bullosa (RDEB)
A phase 3 trial demonstrates prademagene zamikeracel significantly accelerates healing and reduces pain in large, chronic RDEB wounds, offering a promising gene therapy with a favorable safety profile.
Trimethoprim–Sulfamethoxazole in Pregnancy: No Impact on Birth Weight in a Zimbabwean RCT
A large randomized trial in Zimbabwe found that trimethoprim–sulfamethoxazole prophylaxis during pregnancy did not significantly increase birth weight or reduce adverse outcomes.
Inhaled Isoflurane: A Noninferior Alternative to Midazolam for Pediatric ICU Sedation
A multicenter phase 3 trial finds inhaled isoflurane as effective as intravenous midazolam for sedation in mechanically ventilated children, with added benefits of reduced opioid use and faster extubation.
Sweetener Consumption and Genetic Predisposition: New Insights into Early Puberty Risk in Children
Sweetener intake, especially aspartame and sucralose, is linked to increased risk of early puberty in genetically predisposed children, with notable sex-specific differences
Aluminum-Adjuvanted Vaccines in Early Childhood and Risk of Chronic Autoimmune, Allergic, and Neurodevelopmental Disorders: Insights from a Danish Nationwide Cohort Study
A comprehensive Danish cohort study finds no evidence linking early childhood aluminum-adsorbed vaccines to increased risks of autoimmune, allergic, or neurodevelopmental disorders, supporting vaccine safety.
FDA Approves Sephience: A New Oral Therapy for Phenylketonuria
The FDA has approved Sephience, an oral therapy for phenylketonuria (PKU), offering a novel treatment option for both children and adults with this rare metabolic disorder.
EMA Recommends First Oral On-Demand Therapy for Hereditary Angioedema: A Paradigm Shift in Acute Attack Management
EMA endorses sebetralstat, the first oral on-demand therapy for hereditary angioedema, following positive results from the KONFIDENT phase 3 trial, offering a more convenient and rapid treatment option for acute attacks.
Is There a Norovirus Vaccine on the Horizon? Scientific Progress, Challenges, and Future Directions
Norovirus vaccines show promise in clinical trials, but scientific and logistical challenges remain. No approved vaccine exists yet, though novel approaches, including oral pills, are advancing development.
Influenza-Associated Acute Necrotizing Encephalopathy in US Children
A multicenter US study highlights the devastating burden of influenza-associated acute necrotizing encephalopathy in children, with high mortality and disability rates despite aggressive therapy, underscoring the urgent need for prevention
Muscle Strength as a Key Determinant of Metabolic Risk in Paediatric Obesity: New Evidence from Milan
Stronger leg muscles are linked to significantly lower risk of metabolic syndrome in children and adolescents with obesity, highlighting the importance of muscular fitness in paediatric metabolic health.
FDA Expands Skytrofa Approval to Adults with Growth Hormone Deficiency: Clinical and Practice Implications
Once-weekly lonapegsomatropin (Skytrofa) is now FDA-approved for adult growth hormone deficiency, offering a more convenient alternative to daily injections with demonstrated efficacy and safety.
Antibiotics and Pregnancy: Risks of TMP-SMX in Infant Birth Defects
Research highlights increased birth defect risks linked to TMP-SMX use in pregnancy, urging cautious antibiotic selection for treating UTIs.
Severe Childhood Obesity Linked to Elevated Health Risks
A new study highlights the growing prevalence of extreme pediatric obesity in the U.S. and its association with serious metabolic and cardiovascular health risks, emphasizing the need for targeted interventions.
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