A5059696989- Pharmaceutical studies and practices
- Pregnancy and Medication Impact
- Health Systems, Economic Evaluations, Quality of Life
- Pharmaceutical Economics and Policy
- Pediatric Pain Management Techniques
- Child and Adolescent Health
- Maternal Mental Health During Pregnancy and Postpartum
- Statistical Methods in Clinical Trials
- Ion Transport and Channel Regulation
- Breastfeeding Practices and Influences
- Renal Diseases and Glomerulopathies
- Chronic Kidney Disease and Diabetes
- Genomics and Rare Diseases
- Pharmacogenetics and Drug Metabolism
- Pharmacological Effects and Toxicity Studies
- Adolescent and Pediatric Healthcare
- Anesthesia and Sedative Agents
- Metabolism and Genetic Disorders
- Hormonal Regulation and Hypertension
- Reproductive System and Pregnancy
- Ethics and Legal Issues in Pediatric Healthcare
- Cystic Fibrosis Research Advances
- Pregnancy and preeclampsia studies
- Anesthesia and Neurotoxicity Research
- Renin-Angiotensin System Studies
Center for Drug Evaluation and Research
2016-2025
United States Food and Drug Administration
2016-2025
Scripps Clinic
2021
University of Florida
2016-2020
Columbus Oncology and Hematology Associates
2016-2020
Children's National
2018
Reckitt Benckiser (United States)
2016
Inova Children's Hospital
2009
Walter Reed Army Institute of Research
2000
Georgetown University
1995-1998
The therapeutic approach to childhood nephrotic syndrome is based on a series of studies that began with an international collaborative effort sponsored by the International Study Kidney Disease in Children 1967. characteristics children presenting have changed over recent decades greater frequency challenging condition focal segmental glomerulosclerosis and prevalence obesity diabetes mellitus, which may be resistant glucocorticoids former exacerbated long-term glucocorticoid therapy latter...
The authors declared no conflict of interest.
The US Food and Drug Administration (FDA) the European Medicines Agency (EMA) have robust collaboration dialogue around need for data inclusion of pregnant lactating individuals in clinical trials. Despite this collaboration, two agencies their own standards format content labeling these populations. To understand differences, pregnancy lactation sections 31 approved drugs were compared, trends assessed use language concordance discordance related to during between 2 agencies. Further...
The objective of this study was to evaluate the predictive performance population models predict renal clearance in newborns and infants. Pharmacokinetic (PK) data from eight drugs 788 infants were used based on postmenstrual age (PMA), postnatal age, gestational body weight. For PMA model, average fold error for (CL)predicted /CLobserved within a twofold range each drug all subgroups. with > 90% elimination, prediction bias ranged 0.7-1.3. 60-80% 0.6-2.0. Our results suggest that PMA-based...
Two dopamine D1-like receptors have been cloned from mammals, the D1 and D5 receptors, also known as D1A D1B respectively, in rodents. Although are to stimulate renin release, receptor subtype mediating this action has not determined. We investigated expression rat juxtaglomerular cells obtained after enzymatic dispersion of kidney cortex differential centrifugation. Juxtaglomerular primary culture were immunocytochemically 85% 95% positive. These expressed but (mRNA protein). function was...
D2-like receptors in the kidney have been suggested to be important regulation of renin release but subtype(s) expressed juxtaglomerular (JG) cells is not known. Therefore, we determined which family dopamine located primary cultures rat cells.
-Dopamine, via D1-like receptors, stimulates the activity of both protein kinase A (PKA) and C (PKC), which results in inhibition renal sodium transport. Since receptors differentially regulate transport normotensive hypertensive rats, they may also PKC expression these rat strains. Thus, 2 different agonists (fenoldopam or SKF 38393) were infused into artery anesthetized Wistar-Kyoto rats (WKY) spontaneously (SHR) (n=5 to 6/drug/strain). Ten 60 minutes after starting agonist infusion,...
Pompe disease is a lysosomal storage disorder caused by deficiency in the enzyme acid α-glucosidase (GAA). characterized accumulation of glycogen, predominantly muscle tissue, leading to progressive weakness, loss motor, respiratory, and, infantile-onset form, cardiac function. Disease progression highly variable depending on phenotype, but premature death due respiratory complications occurs most patients. Beginning 2006, approved alglucosidase alfa replacement therapies [recombinant human...
The selection of appropriate endpoints in pediatric drug development trials is a critical aspect trial design. Given the high failure rate, selecting optimal design elements, such as primary efficacy endpoint, essential to ensuring increased potential for success. objectives this study were identify measured submitted US Food and Drug Administration relate endpoint attributes label outcome. analysis included pivotal studies from September 2007 July 2016 which there was corresponding adult...
Despite legislation incentivizing and requiring drug companies to conduct pediatric clinical trials, there still is a 9-year delay in approval for labeling after the initial adult approval. The aim of this study was review experience US Food Drug Administration (FDA) with combined trials as means expediting labeling. Combined submitted FDA from 2012 2018 were reviewed. Only publicly available labels reviews utilized analysis. identified 72 products, total 156 trials. therapeutic areas...
The underrepresentation of pregnant individuals in clinical trials and drug development programs causes a lack safety efficacy data for this population poses significant public health challenge. This article outlines various stakeholder efforts that aim to address disparity. It emphasizes the largely untapped potential model-informed (MIDD) tools underscores importance early engagement between pharmaceutical companies regulatory agencies during development. For decades, administration...
Exposure–response (E–R) modeling provides a quantitative tool to leverage adult data support pediatric trial design and drug approval. The E–R studies submitted US Food Drug Administration (FDA) between 2007 2018 were surveyed in the context of various types designs supporting approval population. applications evaluation development programs are mainly focused on three areas: (i) extrapolation when relationships similar populations; (ii) dose selection balance risk–benefit profile based...