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Volume 67(7); Jul 2024
Review Articles
Genetics and Metabolism
Development of orphan drugs for rare diseases
Han-Wook Yoo
Clin Exp Pediatr. 2024;67(7):315-327.   Published online June 28, 2023
· Orphan disease is a rare disease, primarily affecting newborn and children. Vast majority of orphan diseases has genetic background.
· Orphan disease is individually rare. But as a whole, it is not rare, becoming a great socioeconomic burden.
· The diagnosis of rare genetic disease has been problematic, but recent progress of genome analysis technologies makes it faster and more precise.
· There are many unmet needs as to the curative treatment. However, the number of treatable rare diseases is growingly increasing owing to the development of biotechnology.
· Most orphan drugs are extremely expensive because of numer ous hurdles during the process of drug development as well as small number of patients.
Endocrinology
Association between pre- and postnatal exposure to endocrine-disrupting chemicals and birth and neurodevelopmental outcomes: an extensive review
Ozge Yesildemir, Mensure Nur Celik
Clin Exp Pediatr. 2024;67(7):328-346.   Published online November 16, 2023
· Sensitivity to endocrine-disrupting chemical (EDC) exposure increases during critical developmental periods (in embryos, fetuses, and neonates).
· Pre- and postnatal exposure to EDCs is associated with fetal growth restriction, preterm birth, and low birth weight.
· Exposure to EDCs during fetal and early postnatal life can have lasting and lifelong neurodevelopmental outcomes, including autism spectrum, attention deficit hyperactivity, and other cognitive and behavioral disorders.
General Pediatrics
Metabolic complications of obesity in children and adolescents
Hyunjin Park, Jung Eun Choi, Seunghee Jun, Hyelim Lee, Hae Soon Kim, Hye Ah Lee, Hyesook Park
Clin Exp Pediatr. 2024;67(7):347-355.   Published online November 16, 2023
· Pediatric obesity increases the risk of metabolic complications (insulin resistance, dyslipidemia, nonalcoholic fatty liver disease) and long-term cardiovascular diseases.
· A new obesity definition and various indicators (continuous metabolic syndrome score, pediatric simple metabolic syndrome score, fatty liver index) have been proposed to evaluate children’s susceptibility to metabolic disorders.
· Laboratory and body composition tests in pediatric screenings can identify groups at high risk of metabolic complications of obesity.
Editorial
Genetics and Metabolism
Advancing orphan drug development for rare diseases
Jung Min Ko
Clin Exp Pediatr. 2024;67(7):356-357.   Published online November 17, 2023
· Rare diseases present unique challenges and unmet needs for which the development of orphan drugs tailored to them offers hope.
· Despite the hurdles posed by limited patient populations, orphan drug designations from regulatory agencies provide incentives, such as extended market exclusivity and tax credits, that ignite transformative advances.
· Scientific progress in genomics, personalized medicine, and analytics empowers precise interventions by decoding genetic anomalies and encouraging effective treatments.
Original Article
Nutrition
Parental support and exclusive breastfeeding at 3 months in West Java, Indonesia: a mixed-methods approach
Ratu Ayu Dewi Sartika, Fadila Wirawan, Wawan Gunawan, Primasti Nuryandari Putri, Nurul Husna Mohd Shukri
Clin Exp Pediatr. 2024;67(7):358-367.   Published online June 21, 2024
Question: Does paternal support affect exclusive breastfeeding failure?
Finding: Exclusive breastfeeding failure by 3 months was affected by paternal support.
Meaning: Fathers should be included in breastfeeding education and antenatal care.
Letter to the Editor
Developmental and Behavioral Medicine
Efficacy of social skills group intervention among children with mild autism spectrum disorder
Lee Ling Low, Ker Yang Chua, Bih Hwa Ching
Clin Exp Pediatr. 2024;67(7):368-370.   Published online May 31, 2024