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2009
"The aim of this project was to develop a financial model for valuing economic loss caused by neglected diseases prevalent in developing countries. Treatments... 
Rare diseases | economics | Delivery of Health Care | Neglected Diseases | Medical economics
Web Resource
Journal Article
Journal Article
QJM-AN INTERNATIONAL JOURNAL OF MEDICINE, ISSN 1460-2725, 03/2014, Volume 107, Issue 3, pp. 241 - 245
Journal Article
by Hsu, JC and Wu, HC and Feng, WC and Chou, CH and Lai, ECC and Lu, CY
PLOS ONE, ISSN 1932-6203, 09/2018, Volume 13, Issue 9, p. e0204206
Background High-cost orphan drugs are becoming increasingly available to treat rare diseases that affect a relatively small population. Little attention has... 
BUDGET IMPACT | MULTIDISCIPLINARY SCIENCES | ORPHAN DRUGS | Rare diseases | Economic aspects | Care and treatment | Risk factors | Medical care, Cost of
Journal Article
European Journal of Clinical Nutrition, ISSN 0954-3007, 01/2018, Volume 72, Issue 1, pp. 87 - 92
BACKGROUND/OBJECTIVES: Phenylketonuria (PKU) and several other inherited metabolic diseases (IMD) require a lifelong low-protein diet (LPD), otherwise they... 
EUROPE | ADULT PATIENTS | IMPACT | NUTRITION & DIETETICS | MANAGEMENT | GUIDELINES | PHENYLALANINE | FOODS | BURDEN | Caregivers | Rare Diseases - diet therapy | Phenylketonurias - diet therapy | Humans | Child, Preschool | Cost of Illness | Male | Metabolism, Inborn Errors - economics | Diet, Protein-Restricted - economics | Intellectual Disability - prevention & control | Young Adult | Patient Compliance - ethnology | Metabolism, Inborn Errors - ethnology | Adult | Female | Adolescent Nutritional Physiological Phenomena - ethnology | Child | Child Nutritional Physiological Phenomena - ethnology | Food Supply - economics | Intellectual Disability - ethnology | Rare Diseases - ethnology | Rare Diseases - physiopathology | Costs and Cost Analysis | Phenylketonurias - ethnology | Self Report | Rare Diseases - economics | Phenylketonurias - economics | Metabolism, Inborn Errors - diet therapy | Phenylketonurias - physiopathology | Czech Republic | Adolescent | Diet, Protein-Restricted - ethnology | Intellectual Disability - economics | Intellectual Disability - etiology | Metabolism, Inborn Errors - physiopathology | Economic aspects | Complications and side effects | Low-protein diet | Phenylketonuria | Health aspects | Energy intake | Carbohydrates | Compliance | Complications | Patients | Proteins | Nutrition research | Diet | Energy value | Low protein diet | Metabolic disorders | Food
Journal Article
Science Translational Medicine, ISSN 1946-6234, 02/2015, Volume 7, Issue 276, pp. 276ps3 - 276ps3
The portfolio of the National Center for Advancing Translational Sciences (NCATS) rare-diseases therapeutic development program comprises 28 research projects... 
MEDICINE, RESEARCH & EXPERIMENTAL | CELL BIOLOGY | Rare Diseases - economics | Calibration | Research Support as Topic - economics | Translational Medical Research - economics | Healthcare Financing | Humans
Journal Article
Value in Health, ISSN 1098-3015, 05/2018, Volume 21, Issue 5, pp. 525 - 531
Objectives: To review recent studies reporting health care expenditures (budgetary impact) for orphan medicinal products (OMPs) in Europe and to contribute to... 
rare diseases | prevalence | ultrarare diseases | orphan medicinal products | drug prices | budgetary impact | Health Care Costs | Europe | Health Expenditures - statistics & numerical data | Humans | Rare Diseases - drug therapy | Orphan Drug Production - economics | Rare Diseases - economics | Models, Statistical | Budgets
Journal Article