Factors Contributing to the Efficacy-Effectiveness Gap in the Case of Orphan Drugs for Metabolic Diseases.

abstract：:Coronavirus disease 2019 (COVID-19), caused by severe acute respiratory syndrome coronavirus 2 (SARS-CoV‑2), is now a global pandemic. This virus primarily affects the respiratory tract and causes lung injury characterized by acute respiratory distress syndrome. Although the pathophysiology of COVID-19 is not yet clea...

journal_title：Drugs

authors： Hajra A,Mathai SV,Ball S,Bandyopadhyay D,Veyseh M,Chakraborty S,Lavie CJ,Aronow WS

更新日期：2020-10-01 00:00:00

abstract：:Ciclesonide nasal spray delivers the corticosteroid ciclesonide as a hypotonic spray via a metered-dose manual pump. Systemic exposure to ciclesonide and its active metabolite desisobutyryl-ciclesonide is low after intranasal administration. High protein binding (approximately 99%) and rapid first-pass clearance furth...

journal_title：Drugs

authors： Dhillon S,Wagstaff AJ

更新日期：2008-01-01 00:00:00

abstract：:Pain is associated with emotional and physical suffering that severely impacts quality of life. Many guidelines for the treatment of moderate to severe cancer pain indicate the use of opioids. For a small proportion of the global population, opioids are readily accessible, but are consequently also subject to risk of ...

journal_title：Drugs

authors： Marchetti Calônego MA,Sikandar S,Ferris FD,Moreira de Barros GA

更新日期：2020-08-01 00:00:00

abstract：:Bisoprolol is a beta 1-adrenoceptor antagonist with no partial agonist (intrinsic sympathomimetic) activity or membrane stabilising (local anaesthetic) activity. The oral bioavailability of bisoprolol is high (90%) and the drug has a long elimination half-life which allows once-daily administration; in addition, it is...

journal_title：Drugs

authors： Lancaster SG,Sorkin EM

更新日期：1988-09-01 00:00:00

abstract：:Currently available quinolones are either inactive or marginally active against anaerobic bacteria. This review summarises the in vitro activity of currently available as well as experimental quinolones against clinically significant anaerobic bacteria. Quinolones with low activity against anaerobes include ciprofloxa...

journal_title：Drugs

authors： Appelbaum PC

更新日期：1995-01-01 00:00:00

abstract：:Hypertension is common in chronic renal disease and is a risk factor for the faster progression of renal damage, and reduction of blood pressure (BP) is an efficient way of preventing or slowing the progression of this damage. International guidelines recommend lowering BP to 140/90 mm Hg or less in patients with unco...

journal_title：Drugs

authors： Wenzel RR

更新日期：2005-01-01 00:00:00

abstract：:Alectinib (Alecensa(®)) is a second-generation, orally active, potent and highly selective inhibitor of anaplastic lymphoma kinase (ALK). Alectinib is approved for the treatment of ALK fusion-gene positive, unresectable, advanced or recurrent non-small cell lung cancer (NSCLC) in Japan, where it has been given orphan ...

journal_title：Drugs

authors： McKeage K

更新日期：2015-01-01 00:00:00

abstract：:A new formulation of paclitaxel, 130-nanometre albumin-bound paclitaxel (nab-paclitaxel), solubilises hydrophobic paclitaxel and may increase paclitaxel delivery to tumour cells. Intravenous nab-paclitaxel 260 mg/m(2) had a higher maximum whole-blood concentration, shorter time to peak concentration, larger distributi...

journal_title：Drugs

authors： Robinson DM,Keating GM

更新日期：2006-01-01 00:00:00

abstract：:The bacteria most commonly responsible for early-onset (materno-fetal) infections in neonates are group B streptococci, enterococci, Enterobacteriaceae and Listeria monocytogenes. Coagulase-negative staphylococci, particularly Staphylococcus epidermidis, are the main pathogens in late-onset (nosocomial) infections, es...

journal_title：Drugs

authors： Fanos V,Dall'Agnola A

更新日期：1999-09-01 00:00:00

abstract：:After surgery, patients try to minimise discomfort by various manoeuvres including change of posture, immobilisation of injured areas and use of analgesic drugs. The characteristic finding with drug use, revealed by permitting patients to titrate themselves with analgesic from a machine, is that the interindividual do...

journal_title：Drugs

authors： Bullingham RE

更新日期：1985-04-01 00:00:00

abstract：:Pentamidine is an aromatic diamidine derivative which has become one of the standard therapies for Pneumocystis carinii pneumonia (PCP), particularly in patients with acquired immunodeficiency syndrome (AIDS). However, with parenteral administration of the drug there is a high risk of toxicity. Inhaled pentamidine pro...

journal_title：Drugs

authors： Monk JP,Benfield P

更新日期：1990-05-01 00:00:00

abstract：:Healthcare systems are in flux throughout the world. Traditional structures and attitudes are changing. The balance of power between political bodies, payers, providers and patients is being destabilised. New approaches by governments and forward integration by drug companies and payers into care management are all ma...

journal_title：Drugs

authors： Weber F

更新日期：1996-01-01 00:00:00

abstract：:HMG-CoA reductase inhibitors (statins) have been shown to reduce mortality and cardiovascular morbidity in patients with hyperlipidaemia and those with coronary artery disease. However, evidence for statin treatment in patients with chronic heart failure (CHF) remains a subject of debate. Patients with heart failure w...

journal_title：Drugs

authors： Laufs U,Custodis F,Böhm M

更新日期：2006-01-01 00:00:00

abstract：:Capecitabine is an orally administered fluoropyrimidine carbamate used for the treatment of paclitaxel- or anthracycline-refractory breast cancer. Capecitabine is metabolised via a 3-step process to the active agent fluorouracil. The final step of this process occurs preferentially in malignant tissue. In patients wit...

journal_title：Drugs

authors： Dooley M,Goa KL

更新日期：1999-07-01 00:00:00

abstract：:Acute infectious diarrhoea is the major killer of children in the Third World. It is also the principal cause of malnutrition in children under 5 years of age. More than 4 million children die each year of diarrhoea and its complications. Acute diarrhoea kills by causing dehydration, which leads to hypovolaemia and me...

journal_title：Drugs

authors： Gold R

更新日期：1988-01-01 00:00:00

abstract：:Dapoxetine, a selective serotonin reuptake inhibitor, is the first oral pharmacological agent indicated for the treatment of men aged 18-64 years with premature ejaculation. In four randomized, double-blind, placebo-controlled, multicentre studies of 12-24 weeks' duration, oral dapoxetine 30 or 60 mg (administered as ...

journal_title：Drugs

authors： Hoy SM,Scott LJ

更新日期：2010-07-30 00:00:00

abstract：:Colloid solutions have been developed and used over the past 70 years as expanders of the intravascular space, based on an understanding of Starling's law. Increasing osmotic pressure with colloidal products has remained an attractive theoretical premise for volume resuscitation. Indeed, colloids have been shown to in...

journal_title：Drugs

authors： Roberts JS,Bratton SL

更新日期：1998-05-01 00:00:00

abstract：:Subcutaneous dulaglutide (Trulicity®) is a once-weekly glucagon-like peptide-1 receptor agonist that is approved in numerous countries as an adjunct to diet and exercise for the treatment of adults with type 2 diabetes (T2D). In the clinical trial and real-world settings, once-weekly subcutaneous dulaglutide, as monot...

journal_title：Drugs

authors： Scott LJ

更新日期：2020-02-01 00:00:00

abstract：:Chlamydia trachomatis infections are exceedingly prevalent, and can be associated with significant sequelae. The major infections are urethritis, cervicitis, salpingitis, and ocular infection. Chlamydial genital infections present as syndromes, where C. trachomatis is one of the causes of the syndrome. Because specifi...

journal_title：Drugs

authors： Bowie WR

更新日期：1984-05-01 00:00:00

abstract：:Oxatomide is an orally active H1-histamine receptor antagonist which, as appears to occur with some other antihistamines, also inhibits mast cell degranulation. Oxatomide has demonstrated response rates similar to those with other more established members of its drug class in a few studies of chronic urticaria and all...

journal_title：Drugs

authors： Richards DM,Brogden RN,Heel RC,Speight TM,Avery GS

更新日期：1984-03-01 00:00:00

abstract：:Diabetes mellitus has become the most common single cause of end-stage renal disease in the Western world. Diabetic nephropathy, as most forms of renal disease leading to end-stage renal failure, is frequently complicated by arterial hypertension. In type 1 diabetes mellitus the reported excess in arterial hypertensio...

journal_title：Drugs

authors： Viberti G

更新日期：2003-01-01 00:00:00

abstract：:The most important aspect of modern management of acute diarrhoeal illness in children is that of oral rehydration therapy, and drug therapy is very rarely indicated. Despite the dramatic decline in mortality and morbidity in recent years, there is still the need for continuing education in the appropriate use of oral...

journal_title：Drugs

authors： Davies A,Jenkins HR

更新日期：1992-07-01 00:00:00

abstract：:Because of inappropriate signals from the volume-regulation system and a reduction in renal blood flow, the kidney is not able to prevent sodium and water retention in chronic congestive heart failure (CHF). A brief summary of normal renal function and renal involvement in CHF is given and a study of renal function in...

journal_title：Drugs

authors： Ljungman S,Laragh JH,Cody RJ

更新日期：1990-01-01 00:00:00

abstract：:This narrative review reports on the pharmacological and pharmacokinetic properties of rotigotine, a non-ergolinic D₃/D₂/D₁ dopamine receptor agonist approved for the treatment of early- and advanced-stage Parkinson's disease (PD) and moderate to severe restless legs syndrome (RLS). Rotigotine is formulated as a trans...

journal_title：Drugs

authors： Elshoff JP,Cawello W,Andreas JO,Mathy FX,Braun M

更新日期：2015-04-01 00:00:00

abstract：:Serious hepatotoxicity is uncommon with the proper therapeutic use of non-narcotic analgesics but experience with new non-steroidal anti-inflammatory drugs (NSAIDs) is limited. Drugs such as ibufenac, fenclofenac and benoxaprofen were withdrawn from the market because of hepatotoxicity, and liver damage has been repor...

journal_title：Drugs

authors： Prescott LF

更新日期：1986-01-01 00:00:00

abstract：:In this paper, several clinical problems associated with the diagnosis of hypertension are discussed. Blood pressure variability and reactivity are factors underlying the difficulties in the diagnosis of hypertension. These phenomena are interrelated and mixed. White coat hypertension (WCH), referring to the phenomeno...

journal_title：Drugs

authors： Ocón-Pujadas J,Mora-Maciá J

更新日期：1993-01-01 00:00:00

abstract：:Health behaviors such as retention in HIV medical care and adherence to antiretroviral therapy (ART) pose major challenges to reducing new HIV infections, addressing health disparities, and improving health outcomes. Andersen's Behavioral Model of Health Service Use provides a conceptual framework for understanding ho...

journal_title：Drugs

authors： Holtzman CW,Brady KA,Yehia BR

更新日期：2015-04-01 00:00:00

abstract：:The incidence of adenocarcinoma of the pancreas has risen steadily over the past 4 decades. Since pancreatic cancer is diagnosed at an advanced stage, and because of the lack of effective therapies, the prognosis of such patients is extremely poor. Despite advances in our understanding of the molecular biology of panc...

journal_title：Drugs

authors： Rosenberg L

更新日期：2000-05-01 00:00:00

abstract：:Since their introduction 15 years ago, retinoids have been increasingly used for topical and systemic treatment of psoriasis and other hyperkeratotic and parakeratotic skin disorders, keratotic genodermatoses, severe acne and acne-related dermatoses, and also for therapy and/or chemoprevention of skin cancer and other...

journal_title：Drugs

authors： Orfanos CE,Zouboulis CC,Almond-Roesler B,Geilen CC

更新日期：1997-03-01 00:00:00

abstract：:On the basis of well documented biochemical and pharmacological data about the influence of drug mediated enzyme induction on the biotransformation of natural and synthetic sex steroids, practical consequences for hormonal steroid contraception are described and discussed. Clinical reports dealing with this problem ar...

journal_title：Drugs

authors： Hempel E,Klinger W

更新日期：1976-12-01 00:00:00