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Review Article
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.
Allergy
Trends of vitamin D in asthma in the pediatric population for two decades: a systematic review
Myongsoon Sung
Clin Exp Pediatr. 2023;66(8):339-347.   Published online June 14, 2023
· Vitamin D may affect asthma via multiple mechanisms, including lung and optimal immune system functions.
· Many clinical trials have demonstrated the beneficial effects of vitamin D on asthma onset and aggravation. However, definitive clinical trials are lacking, and reports have detailed contradictory effects of vitamin D in children with asthma.
· Some exciting reports stated that obesity and vitamin D deficiency are associated with increased asthma symptoms in the pediatric population.
Understanding noninferiority trials
Seokyung Hahn
Clin Exp Pediatr. 2012;55(11):403-407.   Published online November 23, 2012

Noninferiority trials test whether a new experimental treatment is not unacceptably less efficacious than an active control treatment already in use. With continuous improvements in health technologies, standard care, and clinical outcomes, the incremental benefits of newly developed treatments may be only marginal over existing treatments. Sometimes assigning patients to a placebo is unethical. In such circumstances, there has been...

Acute lymphoblastic leukemia in children: past, present and future
Hyoung Jin Kang, Hee Young Shin, Hyo Seop Ahn
Clin Exp Pediatr. 2007;50(7):601-605.   Published online July 15, 2007
The cure rate of acute lymphoblastic leukemia (ALL) in children dramatically improved over past 5 decades from zero to about 80%. The main cause of improvement is owing to the development of chemotherapy by multicenter clinical trial of large study groups with the understanding of leukemia biology. Recently, pediatric ALL protocols were applied to the treatment of adolescent and even...
Original Article
Clinical Trial of Domestically Developed Bovine Lung Surfactant YY-38 in Neonatal Respiratory Distress Syndrome
Chul Lee, Jeong Nyun Kim, Min-Soo Park, Moon Sung Park, Shin Won Yoon, Wook Chang, Ran Namgung, Kook In Park, Dong-Gwan Han
Clin Exp Pediatr. 1999;42(4):472-483.   Published online April 15, 1999
Purpose : We previously reported modified bovine lung surfactant YY-38(NewfactanⓇ) had a low surface tension, good hysteresis, and exhibited good pressure-volume curve in animal experiment (J Korean Pediatr Asso 1997;40:771-85). We performed multicenter clinical trial of NewfactanⓇ in neonatal RDS. Methods : Seventy-seven infants with RDS(GA 31.8¡¾2.9 wks and BW 1,809¡¾592 gm) in 4 NICU were enrolled. After administration of...