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13:27 EST 12th December 2018 | BioPortfolio

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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Showing "InDex Pharmaceuticals Receives Orphan drug Designation Pediatric Ulcerative" PubMed Articles 1–25 of 18,000+

A composite disease activity index for early drug development in ulcerative colitis: development and validation of the UC-100 score.

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.

Chronological Review of Endoscopic Indices in Inflammatory Bowel Disease.

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...

Dose adjustment in orphan disease populations: the quest to fulfill the requirements of physiologically based pharmacokinetics.

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 ...

Association between Higher Body Mass Index and Pouch-Related Complications during Restorative Proctocolectomy in Patients with Ulcerative Colitis.

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.

5-ASA to sulfasalazine drug switch program in patients with ulcerative colitis.

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.

Enteric Virome and Bacterial Microbiota in Children with Ulcerative Colitis and Crohn's Disease.

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).

Assessing the Correlation of Fecal Calprotectin and the Clinical Disease Activity Index in Patients With Ulcerative Colitis.

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...

From Oxiranes to Oligomers: Architectures of US FDA Approved Pharmaceuticals Containing Oxygen Heterocycles.

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...

Dose-Finding Studies Among Orphan Drugs Approved in the EU: A Retrospective Analysis.

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...

FDA Expectations for Demonstrating Interchangeability.

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...

Performance of the Pediatric Index of Mortality 3 Score in PICUs in Argentina: A Prospective, National Multicenter Study.

To assess the performance of the Pediatric Index of Mortality 3 score in a population of children admitted to PICUs in Argentina.

Completion Rate and Reporting of Mandatory Pediatric Postmarketing Studies Under the US Pediatric Research Equity Act.

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.

Gut microbial and metabolomic profiles after fecal microbiota transplantation in pediatric ulcerative colitis patients.

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 ...

Management of Paediatric Ulcerative Colitis, Part 1: Ambulatory Care- an Evidence-Based Guideline from ECCO and ESPGHAN.

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 Fragility of Statistically Significant Results in Pediatric Orthopaedic Randomized Controlled Trials as Quantified by the Fragility Index: A Systematic Review.

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...

Validation and Investigation of the Operating Characteristics of the Ulcerative Colitis Endoscopic Index of Severity.

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...

Validation of the "German Inflammatory Bowel Disease Activity Index (GIBDI)": An Instrument for Patient-Based Disease Activity Assessment in Crohn's Disease and Ulcerative Colitis.

 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.

Specialty Drug Coverage Varies Across Commercial Health Plans In The US.

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 ...

Adverse Drug Event-Related Admissions to a Pediatric Emergency Unit.

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.

Insights into the Pharmaceuticals and Mechanisms of Neurological Orphan Diseases: Current Status and Future Expectations.

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...

Duration of Pediatric Clinical Trials Submitted to the US Food and Drug Administration.

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.

Outcomes following two-stage surgical approaches in the treatment of pediatric ulcerative colitis.

Surgery for the treatment of ulcerative colitis (UC) can be performed in one-, two-, or three-stage procedures [1]. 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) [1]. An alternative is an initial subtotal colectomy and end ileostomy [2]. In this alternative approach (NIPAA), a completion proctectomy and definitive ileo pouch-anal anastomosis can be...

State-of-the-Art Review on Physiologically Based Pharmacokinetic Modeling in Pediatric Drug Development.

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...

Second-Look Endoscopy in Hospitalized Severe Ulcerative Colitis: A Retrospective Cohort Study.

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.

First Oral Drug for Moderate-To-Severe Ulcerative Colitis.

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