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InDex Pharmaceuticals Receives Orphan Drug Designation Pediatric Ulcerative PubMed articles on BioPortfolio. Our PubMed references draw on over 21 million records from the medical literature. Here you can see the latest InDex Pharmaceuticals Receives Orphan Drug Designation Pediatric Ulcerative articles that have been published worldwide.
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Combining clinical, endoscopic, and histological data associated with ulcerative colitis disease activity in a composite index could be a more sensitive way to detect efficacy in small numbers of patients during early drug development. Our aim was to derive and externally validate a novel index for this purpose.
Inflammatory bowel disease (IBD) is considered a chronic condition characterized by mucosal or transmural inflammation in the gastrointestinal tract. Endoscopic diagnosis and surveillance in patients with IBD have become crucial. In addition, endoscopy is a useful modality in estimation and evaluation of the disease, treatment results, and efficacy of treatment delivery and surveillance. In relation to these aspects, endoscopic disease activity has been commonly estimated in clinical practices and trials. A...
While the media is engaged and fascinated by the idea of 'Precision Medicine', the nuances related to 'Precision Dosing' seem to be largely ignored. Assuming the 'right drug' is selected, clinicians still need to decide on the 'right dose' for individuals. Ideally, optimal dosing should be studied in clinical trials; however, many drugs on the market lack evidence-based dosing recommendations, and small groups of patients (orphan disease populations) are dependent on local guidance and clinician experience ...
Higher body mass index (BMI) is associated with an increased risk of postoperative complications, but only a few studies have clarified the effect of higher BMI on perioperative outcomes in ulcerative colitis (UC). The -purpose of this study was to evaluate the effect of higher BMI on patients undergoing surgery for UC.
To switch patients with ulcerative colitis (UC) from costlier 5-aminosalicylic acid compounds to sulfasalazine and assess (1) the cost savings, (2) the barriers to switching, and (3) adverse events (AEs) and adherence at 3 months after the drug switch.
We examined the fecal enteric virome and bacterial community composition of children with Crohn's disease (CD), ulcerative colitis (UC), and healthy controls to test the hypothesis that unique patterns of viral organisms and/or presence of bacterial pathogens may be identified that could contribute to the pathogenesis of pediatric inflammatory bowel disease (IBD).
It has been shown that fecal calprotectin can be used to evaluate mucosal inflammation better than using clinical indices and serum markers. The aim of this study was to assess the use of fecal calprotectin for evaluating the disease activity in 2 groups of patients with ulcerative colitis and a control group. The study population consisted of 30 patients with active-phase ulcerative colitis, 30 remission-phase patients, and 30 healthy control patients. After obtaining informed consent, we took blood and fe...
Oxygen heterocycles are the second most common type of heterocycles that appear as structural components of US Food and Drug Administration (FDA) approved pharmaceuticals. Analysis of our database of drugs approved through 2017, reveals 311 distinct pharmaceuticals containing at least one oxygen heterocycle. Most prevalent among these are pyranoses, with furanoses, macrolactones, morpholines, and dioxolanes rounding out the top five. The main body of this perspective is organized according to ring-size, com...
In the development process for new drugs, dose-finding studies are of major importance. Absence of these studies may lead to failed phase 3 trials and delayed marketing authorization. In our study we investigated to what extent dose-finding studies are performed in the case of orphan drugs for metabolic and oncologic indications. We identified all orphan drugs that were authorized until August 1, 2017. European Public Assessment Reports were used to extract the final dose used in the summary of product char...
There is a great interest from global companies who are developing biosimilars to pursue interchangeability designation for commercialization of their products in the US. An interchangeability designation will not only allow the substitutability at the pharmacy level without the intervention of a health care provider but the first sponsor who is able to garner interchangeability designation will also receive 12 months of marketing exclusivity. This paper will highlight our current understanding of FDA expec...
To assess the performance of the Pediatric Index of Mortality 3 score in a population of children admitted to PICUs in Argentina.
Many medicines prescribed to children have not been studied or formally approved for pediatric use. The Pediatric Research Equity Act of 2003 authorized the US Food and Drug Administration (FDA) to require pediatric clinical studies.
Ulcerative colitis is a chronic inflammatory disease of the colon that carries a significant disease burden in children. Therefore, new therapeutic approaches are being explored to help children living with this disease. Fecal microbiota transplantation (FMT) has been successful in some children with ulcerative colitis. However, the mechanism of its therapeutic effect in this patient population is not well understood. To characterize changes in gut microbial and metabolomic profiles after FMT, we performed ...
The contemporary management of ambulatory ulcerative colitis (UC) continues to be challenging with ∼20% of children needing a colectomy within childhood years. We thus aimed to standardize daily treatment of paediatric UC and inflammatory bowel diseases (IBD)-unclassified through detailed recommendations and practice points.
The randomized controlled trial (RCT) is the gold standard study design allowing critical comparison of clinical outcomes while minimizing bias. Traditionally clinical trials are evaluated through statistical significance, expressed by P-values and confidence intervals. However, until recently, the robustness of a study's conclusions has been given little attention. A new metric, the fragility index, quantifies the number of patients theoretically required to switch outcomes in order to reverse the study co...
The Ulcerative Colitis Endoscopic Index of Severity (UCEIS) is a novel instrument to evaluate endoscopic disease activity. It has been demonstrated to outperform the more widely used Mayo endoscopic score (MES) in predicting long-term prognosis, including the need for colectomy. Despite its potential benefits, many clinicians still prefer to use MES because its operating characteristics are better defined and its grades are more readily applicable to clinical decision-making. The aims of our study were to q...
Assessment of disease activity in Crohn's disease (CD) and ulcerative colitis (UC) is usually based on the physician's evaluation of clinical symptoms, endoscopic findings, and biomarker analysis. The German Inflammatory Bowel Disease Activity Index for CD (GIBDI) and UC (GIBDI) uses data from patient-reported questionnaires. It is unclear to what extent the GIBDI agrees with the physicians' documented activity indices.
We analyzed specialty drug coverage decisions issued by the largest US commercial health plans to examine variation in coverage and the consistency of those decisions with indications approved by the Food and Drug Administration (FDA). Across 3,417 decisions, 16 percent of the 302 drug-indication pairs were covered the same way by all of the health plans, and 48 percent were covered the same way by 75 percent of the plans. Specifically, 52 percent of the decisions were consistent with the FDA label, 9 ...
The objectives of this study were to analyze adverse drug events (ADEs) related to admissions to a pediatric emergency unit and to identify the associated risk factors.
Several rare or orphan diseases have been characterized that singly affect low numbers of people, but cumulatively reach ∼6% - 10% of the population in Europe and in the United States. Human genetics has shown to be broadly effective when evaluating subjacent genetic defects such as orphan genetic diseases, but on the other hand, a modest progress has been achieved toward comprehending the molecular pathologies and designing new therapies. Chemical genetics, placed at the interface of chemistry and geneti...
The increasing prevalence of pediatric chronic disease has resulted in increased exposure to long-term drug therapy in children. The duration of recently completed drug trials that support approval for drug therapy in children with chronic diseases has not been systematically evaluated. Such information is a vital first step in forming safety pharmacovigilance strategies for drugs used for long-term therapy in children.
Surgery for the treatment of ulcerative colitis (UC) can be performed in one-, two-, or three-stage procedures . The more traditional approach is a total proctocolectomy and creation of an ileo pouch-anal anastomosis and diverting stoma at the initial operation, followed by ileostomy closure several weeks later (TIPPA) . An alternative is an initial subtotal colectomy and end ileostomy . In this alternative approach (NIPAA), a completion proctectomy and definitive ileo pouch-anal anastomosis can be...
Physiologically based pharmacokinetic modeling and simulation is an important tool for predicting the pharmacokinetics, pharmacodynamics, and safety of drugs in pediatrics. Physiologically based pharmacokinetic modeling is applied in pediatric drug development for first-time-in-pediatric dose selection, simulation-based trial design, correlation with target organ toxicities, risk assessment by investigating possible drug-drug interactions, real-time assessment of pharmacokinetic-safety relationships, and as...
Acute severe ulcerative colitis (ASUC) is a serious complication of ulcerative colitis (UC). Management of partial responders to steroids or rescue therapy remains challenging. Whether there is a role for re-look sigmoidoscopic evaluation in disease management is unknown.