Results 101 to 110 of about 88,754 (289)
Pharmaceutical compounding of orphan active ingredients in Belgium : how community and hospital pharmacists can address the needs of patients with rare diseases [PDF]
, 2019 Background: Pharmaceutical compounding of orphan active ingredients can offer cost-effective treatment to patients when no other drug product is available for a rare disease or during periods of drug product shortages.
Boussery, Koen +7 more
core +2 more sources
British Journal of Clinical Pharmacology, EarlyView.Abstract Aim The aim of this study was to assess the general and product‐specific experiences of MAHs use of RWD/RWE in medicines development and in their regulatory submissions, and to explore organizational aspects of MAHs related to RWD/RWE. Methods An electronic survey was conducted, and information collected directly from MAHs.
Sini M. Eskola +5 more
wiley +1 more source
Book Review: Deforming Tort Reform [PDF]
, 1990 The storms buffeting the tort system over the past two decades have come in three distinct waves. In the late 1960s, steep increases in the insurance costs incurred by health care providers protecting against negligence claims by patients triggered what ...
Page, Joseph A.
core +1 more source
Extending life for people with a terminal illness: a moral right and an expensive death? Exploring societal perspectives [PDF]
, 2015 BACKGROUND: Many publicly-funded health systems apply cost-benefit frameworks in response to the moral dilemma of how best to allocate scarce healthcare resources.
Baker, Rachel M. +7 more
core +4 more sources
Nanomaterial‐based immune therapeutic strategies in neurodegenerative diseases
BMEMat, EarlyView.This review highlights the immunomodulatory potential of nanomaterials (NMs) in treating neurodegenerative diseases (NDs). It focuses on their roles in regulating innate and adaptive immune responses to maintain immune homeostasis. By providing insights into these mechanisms, the review lays the groundwork for innovative NMs therapeutic strategies to ...
Xinru Zhou +6 more
wiley +1 more source
Functional classification of G-Protein coupled receptors, based on their specific ligand coupling patterns [PDF]
, 2006 Functional identification of G-Protein Coupled Receptors (GPCRs) is one of the current focus areas of pharmaceutical research. Although thousands of GPCR sequences are known, many of them re- main as orphan sequences (the activating ligand is unknown ...
Bakır, Burcu, Sezerman, Uğur
core +1 more source
Developmental Dynamics, EarlyView.Abstract Trichorhinophalangeal syndrome (TRPS) is a rare genetic disease inherited in an autosomal dominant manner. It occurs in 1 in 100,000 people globally and is caused by several types of mutations of the TRPS1 gene. Since the first human patient was reported in 1966, typical and atypical pathologies, disease courses, and treatment case ...
Naoya Saeki +6 more
wiley +1 more source
Children in need of medical innovation [PDF]
Children are therapeutic orphans and an underprivileged group in innovations derived from drug therapy. As the innovation process of pharmaceuticals is a long, risky and very costly business, economists typically emphasise lack of profit incentives and ...
Entorf, Horst +2 more
core
Clinical and biochemical improvements in a patient with MNGIE following enzyme replacement. [PDF]
, 2013 Mitochondrial neurogastrointestinal encephalomyopathy (MNGIE) is a rare autosomal recessive metabolic disorder caused by a deficiency of thymidine phosphorylase (TP, EC2.4.2.4) due to mutations in the nuclear gene TYMP.
Bain, MD +5 more
core +1 more source
Orphanet Journal of Rare Diseases, 2012 Since its enactment in 2000, the European Orphan Medicinal Products Regulation has allowed the review and approval of approaching 70 treatments for some 55 different conditions in Europe. Success does not come without a price, however.
Hughes-Wilson Wills +3 more
doaj +1 more source