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PubMed Journals Articles About "InDex Pharmaceuticals Receives Orphan Drug Designation Pediatric Ulcerative" RSS

17:22 EST 23rd November 2017 | 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 17,000+

Direct-to-Consumer Genetic Testing and Orphan Drug Development.

Since the introduction of the Orphan Drug Act (ODA) in 1983, orphan drug approvals in the United States have jumped from


Demonstrating significant benefit of orphan medicines: analysis of 15 years of experience in Europe.

In the European Union demonstration of 'significant benefit' is mandatory if satisfactory methods exist for a disease targeted by a new orphan medicinal product. Significant benefit is required at the time of orphan designation, when it can be supported by preclinical studies, and at the time of marketing authorization, when clinical data are needed. For the first time, our work has identified, defined and organized the scientific grounds on which significant benefit is granted in the European Union, based ...

The Use of Social Media in Orphan Drug Development.

Social media has transformed how people interact with one another through the Internet, and it has the potential to do the same for orphan drug development. Currently, social media influences the orphan drug development process in the following three ways: assisting the study of orphan diseases, increasing the awareness of orphan disease, and playing a vital role in clinical trials. However, there are some caveats to the utilization of social media, such as the need to protect patient privacy by adequately ...


CMV Infection in Pediatric Severe Ulcerative Colitis - A Multicenter Study from the Pediatric IBD Porto Group of ESPGHAN.

Data on the clinical course and outcomes of pediatric patients with cytomegalovirus (CMV) infection complicating acute severe ulcerative colitis (ASC) are very limited. The aim of our study was to compare outcomes of children with ASC who were CMV-positive or CMV-negative.

Pharmacology and drug development in rare diseases: the attractiveness and expertise of the French medical pharmacology.

Developing drugs for rare disease can be challenging due to specific rare disease characteristics. The French Medical Pharmacology is structured and positioned to play a major role in Orphan Drug Research and Development due to the required expertise concentrated into pharmacology departments, exclusively implemented within the French University Hospitals, public hospitals that are linked to a medical school (and often a pharmacy school) with numerous INSERM or CNRS labelled research units. In addition, the...

The Changing Face of Pediatric Ulcerative Colitis: A Population-based Cohort Study.

The aims of this retrospective study were to describe ulcerative colitis (UC) phenotype at diagnosis and follow-up and to identify possible predictors of severe disease course.

Subcutaneous Golimumab in Pediatric Ulcerative Colitis: Pharmacokinetics and Clinical Benefit.

Current treatments for pediatric ulcerative colitis (UC) are limited. We evaluated the pharmacokinetics and clinical benefits of subcutaneous golimumab, an anti-tumor necrosis factor agent, in moderately-to-severely active pediatric patients with UC refractory to conventional therapy.

Fecal Microbial Transplantation for Pediatric Ulcerative Colitis: Are We There Yet?

Drugs for Children.

The "Therapeutic Orphan" status of children is gradually improving, but our efforts to eliminate off-label/off-evidence use must be sustained for safer and more effective pediatric drug therapy. This is even more important in resource-scarce settings, because of the increasing child population in those regions. The juncture of pediatric pharmacology and global child health represents an emerging field of clinical pharmacology illuminating a crucial societal need on a global scale.

Oral delivery of nanoparticles loaded with ginger active compound, 6-shogaol, attenuates ulcerative colitis and promotes wound healing in a murine model of ulcerative colitis.

Oral drug delivery is the most attractive pathway for ulcerative colitis (UC) therapy, since it has many advantages. However, this strategy has encountered many challenges, including the instability of drugs in the gastrointestinal tract (GT), low targeting of disease tissues, and severe adverse effects. Nanoparticles capable of colitis-tissue-targeted delivery and site-specific drug release may offer a unique and therapeutically effective system that addresses these formidable challenges.

Statistical modeling for Bayesian extrapolation of adult clinical trial information in pediatric drug evaluation.

Children represent a large underserved population of "therapeutic orphans," as an estimated 80% of children are treated off-label. However, pediatric drug development often faces substantial challenges, including economic, logistical, technical, and ethical barriers, among others. Among many efforts trying to remove these barriers, increased recent attention has been paid to extrapolation; that is, the leveraging of available data from adults or older age groups to draw conclusions for the pediatric populat...

Histologic Correlates of Clinical and Endoscopic Severity in Children Newly Diagnosed With Ulcerative Colitis.

To characterize rectal histology in an inception cohort of children newly diagnosed with ulcerative colitis (UC) and to explore its relationship with clinical indices of disease severity. The PROTECT (Predicting Response to Standardized Pediatric Colitis Therapy) Study enrolled children 17 years of age and younger newly diagnosed with UC. Baseline rectal biopsies were evaluated for acute and chronic inflammation, eosinophilic inflammation (peak eosinophil count > 32 eosinophils/high powered field, eosinophi...

A Rule of Unity for Human Intestinal Absorption 3: Application to Pharmaceuticals.

The rule of unity is based on a simple absorption parameter, Π, that can accurately predict whether or not an orally administered drug will be well absorbed or poorly absorbed. The intrinsic aqueous solubility and octanol-water partition coefficient, along with the drug dose are used to calculate Π. We show that a single delineator value for Π exist that can distinguish whether a drug is likely to be well absorbed (FA ≥ 0.5) or poorly absorbed (FA < 0.5) at any specified dose. The model is shown to giv...

Ultrasound of the pediatric appendix.

Appendicitis is the most common pediatric surgical emergency. Ultrasound (US) receives the highest appropriate rating scale in children with right lower quadrant pain suspected to have appendicitis. The US exam of the appendix has improved since Puylaert pioneered the technique of graded compression in 1986. In this article, we review ultrasonography of the pediatric appendix as it pertains to the normal appendix, acute appendicitis and the different sonographic manifestations. We also briefly describe tech...

Hepatotoxicity of Herbal Supplements Mediated by Modulation of Cytochrome P450.

Herbal supplements are a significant source of drug-drug interactions (DDIs), herb-drug interactions, and hepatotoxicity. Cytochrome P450 (CYP450) enzymes metabolize a large number of FDA-approved pharmaceuticals and herbal supplements. This metabolism of pharmaceuticals and supplements can be augmented by concomitant use of either pharmaceuticals or supplements. The xenobiotic receptors constitutive androstane receptor (CAR) and the pregnane X receptor (PXR) can respond to xenobiotics by increasing the exp...

Rapid Induction Therapy with Oral Tacrolimus in Elderly Patients with Refractory Ulcerative Colitis Can Easily Lead to Elevated Tacrolimus Concentrations in Blood: A Report of 5 Cases.

BACKGROUND Tacrolimus is reportedly effective for the treatment of refractory ulcerative colitis (UC). At our hospital, there has been an increase in the number of patients, including elderly patients, with refractory UC treated with tacrolimus. Here, we review the data from 5 patients with elderly-onset UC treated with tacrolimus as remission induction therapy. CASE REPORT The subjects were 5 patients ≥65 years of age with refractory UC who had received oral tacrolimus as remission induction therapy betw...

Suspended solids moderate the degradation and sorption of waste water-derived pharmaceuticals in estuarine waters.

This study focuses on the fate of pharmaceuticals discharged into an estuarine environment, particularly into the Turbidity Maximum Zone (TMZ). Batch experiments were set up to investigate the factors regulating the degradation of 53 selected pharmaceuticals. Treated effluents from Bordeaux city (France) were mixed with water from the estuarine Garonne River during 4weeks under 6 characterized conditions in order to assess the influence of suspended particulates, sterilization, untreated wastewater input an...

Effects of exposure to pharmaceuticals (diclofenac and carbamazepine) spiked sediments in the midge, Chironomus riparius (Diptera, Chironomidae).

Human and veterinary pharmaceuticals and degradation products are continuously introduced into the environment. To date, there is a lack of information about the effects of pharmaceuticals in spiked toxicity tests with non-target organisms. In this study, we have evaluated the effects of exposure to two common pharmaceuticals in the midge Chironomus riparius in spiked sediment experiments. The selected pharmaceuticals are the nonsteroidal anti-inflammatory drug (NSAID): diclofenac (DF) and the anti-depressa...

How Secure is the Diagnosis of Ulcerative Colitis in Children, Even After Colectomy?

We reviewed all children who have undergone a colectomy for ulcerative colitis in our tertiary referral centre in a twelve-year period to assess the rate of reclassification as Crohn's disease. In contrast to Crohn's Disease, a colectomy is considered to be definitive treatment for patients with ulcerative colitis. Distinguishing between the two can be challenging when disease is manifest only within the colon - even histological examination of a colectomy specimen may be inconclusive. Historically, the rec...

Treatment Outcomes of Children with HIV Infection and Drug-Resistant TB in Three Provinces in South Africa, 2005-2008.

To describe outcomes of HIV-infected pediatric patients with drug-resistant tuberculosis (DR TB).

Diabetes Drug Receives Boxed Warning for Increased Risk of Leg and Foot Amputations.

Methodological and Ethical Issues in Pediatric Medication Safety Research.

In May 2016, the Eshelman School of Pharmacy at The University of North Carolina at Chapel Hill convened the PharmSci conference to address the topic of "methodological and ethical issues in pediatric medication safety research." A multidisciplinary group of experts representing a diverse array of perspectives, including those of the US Food and Drug Administration, children's hospitals, and academia, identified important considerations for pediatric medication safety research and opportunities to advance t...

Transanal Versus Transabdominal Minimally Invasive (Completion) Proctectomy With Ileal Pouch-anal Anastomosis in Ulcerative Colitis: A Comparative Study.

This study aims to compare surgical outcome of transanal ileal pouch-anal anastomosis (ta-IPAA) with transabdominal minimal invasive approach in ulcerative colitis (UC), using the comprehensive complication index (CCI).

2016 in review: FDA approvals of new molecular entities.

An overview of drugs approved by FDA in 2016 reveals dramatic disruptions in long-term trends. The number of new molecular entities (NMEs) dropped, reflecting the lowest rate of small-molecule approvals observed in almost five decades. In addition, the pace of industry consolidation slowed substantially. The impact of mergers and acquisitions decreased the total number of organizations with past approval experience and continued research and development (R&D) activities to 102, divided evenly between more e...

Golimumab for the treatment of ulcerative colitis.

Tumor necrosis factor antagonists have revolutionized the therapeutic management of inflammatory bowel disease. Infliximab and adalimumab were the first biological agents used to induce and maintain remission in ulcerative colitis. More recently, a third tumor necrosis factor antagonist, golimumab, was approved, extending the therapeutic approach for moderate-to-severe ulcerative colitis. Areas covered: In this review, the authors review the literature on the efficacy and safety of golimumab in the context ...


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