Results 41 to 50 of about 10,049 (144)

Pediatric Developmental Safety Assessment: Are We Ready for the Next Thalidomide?

Clinical Pharmacology &Therapeutics, EarlyView.
Pediatric drug development has achieved remarkable success in the last 20 years with over 1,000 products studied in pediatric patients. This success has been driven in part by an increased understanding of pediatric disease processes. The aspect that has been largely overlooked is the potential adverse effect of new drugs on pediatric developmental ...
Gilbert J. Burckart, Susan M. Abdel‐Rahman, Gelareh Abulwerdi, Sherbet Samuels, Rebecca Racz, Mohamed A. Mohamoud, Nicholas P. Tatonetti   +6 more
wiley   +1 more source

A Semantic Transformation Methodology for the Secondary Use of Observational Healthcare Data in Postmarketing Safety Studies

Frontiers in Pharmacology, 2018
Background: Utilization of the available observational healthcare datasets is key to complement and strengthen the postmarketing safety studies. Use of common data models (CDM) is the predominant approach in order to enable large scale systematic ...
Anil Pacaci, Anil Pacaci, Suat Gonul, Suat Gonul, A. Anil Sinaci, Mustafa Yuksel, Gokce B. Laleci Erturkmen   +6 more
doaj   +1 more source

Balancing benefits and risks in the era of biologics

Therapeutic Advances in Musculoskeletal Disease, 2019
Biologics are substances synthetized from biological sources used in the prevention and treatment of several diseases. Rheumatologists have many years of experience with biologics for the treatment of immune-mediated diseases and osteoporosis. Randomized
Giovanni Adami, Kenneth G. Saag, Roland D. Chapurlat, Nuria Guañabens, Glenn Haugeberg, Willem F. Lems, Radmila Matijevic, Nicola Peel, Denis Poddubnyy, Piet Geusens   +9 more
doaj   +1 more source

A Practical Guide to Implementing Real‐World Evidence as Primary Basis for Approval and Label Expansion

Clinical Pharmacology &Therapeutics, EarlyView.
In June 2026, the FDA issued a revised draft guidance describing the circumstances in which one adequate and well‐controlled clinical investigation plus confirmatory evidence may satisfy the statutory substantial evidence standard for establishing efficacy and safety. Confirmatory evidence includes real‐world evidence (RWE).
Tracy J. Mayne, Maurice A. Brookhart, Nancy A. Dreyer   +2 more
wiley   +1 more source

Prolonged fenfluramine use in open‐label studies of Dravet or Lennox–Gastaut syndromes: Long‐term safety, tolerability, patient global functioning, and considerations for interpreting effectiveness

Epilepsia, EarlyView.
This graphical abstract provides an overview of the content from this open‐label extension study of fenfluramine use in patients with Dravet syndrome or Lennox‐Gastaut syndrome. Abstract Objective Long‐term safety and global functioning are reported in patients with Dravet syndrome (DS) or Lennox–Gastaut syndrome (LGS) treated with fenfluramine in an ...
Antonio Gil‐Nagel, Kelly G. Knupp, Boudewijn Gunning, Domenica Immacolata Battaglia, Elizabeth A. Thiele, Ingrid E. Scheffer, An‐Sofie Schoonjans, Rima Nabbout, Nathalie Villeneuve, Kerstin Alexandra Klotz, Nicola Specchio, Rocio Sanchez‐Carpintero, Marta Zolnowska, Anne‐Liv Schulz, Lily Perrin, Najla Dickson, Mélanie Langlois, Amélie Lothe, Joseph Sullivan   +18 more
wiley   +1 more source

Sucrose-Formulated Recombinant Factor VIII Dosing Flexibility in Prophylaxis Regimens: Experience from Postmarketing Surveillance Studies

Advances in Hematology, 2015
Objectives. Prophylaxis regimens for severe hemophilia A allowing more flexible dosing while maintaining efficacy may improve adherence and decrease the cost of prophylaxis.
Thomas J. Humphries, Stephan Rauchensteiner, Claudia Tückmantel, Alexander Pieper, Monika Maas Enriquez, Prasad Mathew   +5 more
doaj   +1 more source

Antiseizure potency and neurotoxicity of the enantiomers of fenfluramine and norfenfluramine in rats and correlations with their concentrations in plasma and brain

Epilepsia, EarlyView.
Abstract Objective Based on the evidence that fenfluramine‐induced anorexia, weight loss, and cardiovascular toxicity are primarily mediated by the d‐enantiomers of fenfluramine and its metabolite norfenfluramine, we investigated pharmacokinetic/pharmacodynamic correlations for the active enantiomers of fenfluramine and norfenfluramine in the rat ...
Yara Sheeni, Dinorah Barasch, Tawfeeq Shekh‐Ahmad, Emilio Perucca, Meir Bialer   +4 more
wiley   +1 more source

Multi‐omics–driven precision medicine

iMeta, EarlyView.
Multi‐omics‐driven precision medicine (MODPM) provides a multiscale, continuously learnable framework that integrates genomics, epigenomics, transcriptomics, proteomics, metabolomics, microbiome profiles, and clinical data. Powered by artificial intelligence and foundation models, MODPM enables cross‐modal representation learning, contextual modeling ...
Huibo Li, Zhe Zhao, Yifan Zhang, Yao Ma, Gaofei Hu, Min Zeng, Zhanqun Yang, Zixuan Zhao, Xin Zhou, Wei Hu, Yuxuan Sun, Meng Su, Jun Li, Matthew Whiteman, Wei Fu, Chao Zhong, Lemin Zheng, Long Chen, Hairong Lv, Rongsheng Zhao, Yi Zhun Zhu   +20 more
wiley   +1 more source

Collagen‐membrane‐covered matrix‐associated chondrocyte implantation improves safety over periosteum‐covered MACI for large knee cartilage defects: A real‐world data analysis in the registry study

Knee Surgery, Sports Traumatology, Arthroscopy, EarlyView.
Abstract Purpose This registry study aimed to evaluate the safety and effectiveness of collagen membrane‐covered matrix‐associated autologous chondrocyte implantation (cMACI) versus periosteum‐covered MACI (pMACI) for large knee cartilage defects (Outerbridge III–IV, ≥4 cm²) in the femorotibial and/or patellofemoral joints with a minimum 2‐year follow ...
Yuji Uchio, Ryosuke Kuroda, Yasuo Niki, Katsura Sugawara, Yasuyuki Ishibash   +4 more
wiley   +1 more source

Direct oral anticoagulant approvals by four major regulatory agencies: a cross-sectional analysis of premarket and postmarket evidence

BMJ Open
Objectives To compare the premarket and postmarket evidence of safety and efficacy of direct oral anticoagulants approved for stroke prevention in atrial fibrillation patients across four major regulatory agencies.Design Cross-sectional.Setting European ...
Joseph S Ross, Tianna Zhou, Maryam Mooghali   +2 more
doaj   +1 more source