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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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Research in rare diseases has contributed substantially toward the current understanding in the pathophysiology of the common diseases. However, medical needs of patients with rare diseases have always been neglected by the society and pharmaceutical industries based on their small numbers and unprofitability. The Orphan Drug Act (1983) was the first serious attempt to address the unmet medical needs for patients with rare diseases and to provide impetus for the pharmaceutical industry to promote orphan dru...
Though pharmacokinetic studies suggest accelerated biologic drug clearance with increasing body weight, evidence of obesity's impact on clinical outcomes in biologic-treated patients with ulcerative colitis (UC) is inconsistent.
The genetic contributions to pediatric onset ulcerative colitis (UC), characterized by severe disease and extensive colonic involvement, are largely unknown. In adult onset UC, Genome Wide Association Study (GWAS) has identified numerous loci, most of which have a modest susceptibility risk (OR 0.84-1.14), with the exception of the human leukocyte antigen (HLA) region on Chromosome 6 (OR 3.59).
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...
Osteosarcoma is an orphan disease for which little improvement in survival has been made since the late 1980s. New drug discovery for orphan diseases is limited by the cost and time it takes to develop new drugs. Repurposing already approved FDA-drugs can help overcome this limitation. Another limitation of cancer drug discovery is the lack of preclinical models that accurately recapitulate what occurs in humans. For OS using dogs as a model can minimize this limitation as OS in canines develops spontaneous...
The surgical comorbidity assessment is important for patient risk stratification, counseling, and research. In adults, risk assessment indices, such as the Charlson Co-morbidity Score (CCS) or Van Walraven Index (VWI), are well established. In pediatrics, however, risk assessment indices are scarce. Recently, a pediatric-specific risk assessment index, the Rhee index, was developed to discriminate mortality for pediatric general surgery patients. Currently, there is no validated risk assessment tool in pedi...
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.
IV ciclosporin therapy is effective in steroid-refractory ulcerative colitis. The optimal drug level to achieve response and minimize complications during induction therapy is not known.
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.
Vitamin D regulates intestinal epithelial and immune functions, and vitamin D receptor deficiency increases the severity of murine colitis. Bioavailable 25-hydroxyvitamin D (25(OH)D) is available to target tissues and may be a driver of immune function. The aim is to evaluate the relationship of bioavailable 25(OH)D to the clinical expression of treatment naive pediatric ulcerative colitis (UC).
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...
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).
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...
Pediatric ulcerative colitis (UC) presents at an earlier age and increasing prevalence. Our aim was to examine morbidity, steroid sparing strategies, and surgical outcome in children with active UC.
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...
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...
To evaluate the reliability and validity of the Chinese version of pediatric voice handicap index (pVHI).
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 Clinical Trials Transformation Initiative convened with several groups in the pediatric antibacterial drug development community with the goal of identifying challenges and recommending ways to improve current practice. Attention to 5 major areas hold the promise of making new antibiotics available for use in children as soon as possible after they are approved for use in adults.
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 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.
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 ...
To evaluate whether and when Drug-Induced Sleep Endoscopy (DISE) changes diagnosis and treatment plan in pediatric Obstructive Sleep Apnoea Syndrome (OSAS) with the aim to identify specific subgroups of patients for whom DISE should be especially considered.
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.