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Catching Them Early: Framework Parameters and Progress for Prenatal and Childhood Application of Advanced Therapies.

ABSTRACT: Advanced therapy medicinal products (ATMPs) are medicines for human use based on genes, cells or tissue engineering. After clear successes in adults, the nascent technology now sees increasing pediatric application. For many still untreatable disorders with pre- or perinatal onset, timely intervention is simply indispensable; thus, prenatal and pediatric applications of ATMPs hold great promise for curative treatments. Moreover, for most inherited disorders, early ATMP application may substantially improve efficiency, economy and accessibility compared with application in adults. Vindicating this notion, initial data for cell-based ATMPs show better cell yields, success rates and corrections of disease parameters for younger patients, in addition to reduced overall cell and vector requirements, illustrating that early application may resolve key obstacles to the widespread application of ATMPs for inherited disorders. Here, we provide a selective review of the latest ATMP developments for prenatal, perinatal and pediatric use, with special emphasis on its comparison with ATMPs for adults. Taken together, we provide a perspective on the enormous potential and key framework parameters of clinical prenatal and pediatric ATMP application.

SUBMITTER: Lederer CW

PROVIDER: S-EPMC9031205 | biostudies-literature | 2022 Apr

REPOSITORIES: biostudies-literature

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Catching Them Early: Framework Parameters and Progress for Prenatal and Childhood Application of Advanced Therapies.

Lederer Carsten W CW Koniali Lola L Buerki-Thurnherr Tina T Papasavva Panayiota L PL La Grutta Stefania S Licari Amelia A Staud Frantisek F Bonifazi Donato D Kleanthous Marina M

Pharmaceutics 20220405 4

Advanced therapy medicinal products (ATMPs) are medicines for human use based on genes, cells or tissue engineering. After clear successes in adults, the nascent technology now sees increasing pediatric application. For many still untreatable disorders with pre- or perinatal onset, timely intervention is simply indispensable; thus, prenatal and pediatric applications of ATMPs hold great promise for curative treatments. Moreover, for most inherited disorders, early ATMP application may substantia ...[more]

PMID: 35456627

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Project description:BackgroundAntibiotic exposure in pregnancy is associated with reduced microbiome diversity in the infant gut. Given that recent research has shown that early microbiome health can impact child socioemotional development, we examined the relationship between prenatal antibiotic exposure in pregnancy and childhood socioemotional developmental outcomes using a large, nationally representative longitudinal dataset.MethodsA sample of 4800 diverse families were assessed from the population cohort of the Growing Up in New Zealand Study (GUiNZ), which prospectively follows children starting in the last trimester of pregnancy into early childhood. Socioemotional development was measured using a composite score derived from seven commonly used socioemotional tasks administered between 9 months and 4.5 years of child age, addressing emotional expression understanding, regulation of emotions and behavior, and social problem solving and relationship skills. A national comprehensive pharmaceutical database was used to determine children's prenatal antibiotic exposure. Multivariate linear regressions models were used to examine the effects of the timing (trimester) and dosage (number of courses) of prenatal antibiotic exposure on socioemotional development, with and without statistically adjusting for confounding factors addressing maternal health, socioeconomic status, maternal age, and child sex.ResultsIn unadjusted analyses, antibiotic exposure was inversely associated with child socioemotional development. However, after statistically adjusting for important confounds, socioemotional development was not associated with prenatal antibiotic exposure at any dosage or trimester of pregnancy (all β ≤ -0.02).ConclusionPrenatal antibiotic exposure does not appear to impact early childhood socioemotional development. Maternal health and sociodemographic factors are confounded with antibiotic exposure and socioemotional development, a fact that should be considered in future research examining the effects of prenatal antibiotic exposure on child health. These findings may be reassuring to families who are concerned about the long-term effects of antibiotics in pregnancy on child health outcomes.

Dataset Information

Catching Them Early: Framework Parameters and Progress for Prenatal and Childhood Application of Advanced Therapies.

Publications

Catching Them Early: Framework Parameters and Progress for Prenatal and Childhood Application of Advanced Therapies.

Similar Datasets

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