المؤلفون: Grand, Tobias Sydendal^1,2 (AUTHOR) tsgrand1@sheffield.ac.uk, Ren, Shijie¹ (AUTHOR), Hall, James³ (AUTHOR), Åström, Daniel Oudin² (AUTHOR), Regnier, Stephane² (AUTHOR), Thokala, Praveen¹ (AUTHOR)

المصدر: PharmacoEconomics. Jun2024, Vol. 42 Issue 6, p619-631. 13p.

مصطلحات موضوعية: *ECONOMIC models, RARE diseases, ORPHAN drugs, ECONOMIC opportunities, TECHNOLOGY assessment

المؤلفون: SALTONSTALL, PETER¹ paul@kendallsps.com, ROSS, HEIDI¹, KIM, PAUL T.²

المصدر: Milbank Quarterly. Mar2024, Vol. 102 Issue 1, p83-96. 14p.

مصطلحات موضوعية: *DIFFUSION of innovations, *INVESTMENTS, *HEALTH care reform, *TAXATION, ORPHAN drug laws, CRITICISM, ORPHAN drugs, RARE diseases, PATIENT advocacy, DRUG approval, DRUG development, MEDICAL needs assessment, MEDICAL care costs, PATIENT Protection & Affordable Care Act, PATIENTS' attitudes

مصطلحات جغرافية: UNITED States

الشركة/الكيان: UNITED States. Food & Drug Administration

المؤلفون: Ondoño Molina, Raul

المساهمون: University/Department: Universitat Ramon Llull. Departament de Química Orgànica i Farmacèutica

مرشدي الرسالة: Estrada-Tejedor, Roger, Borrell, Jose I.

المصدر: TDX (Tesis Doctorals en Xarxa)

مصطلحات موضوعية: Myotonic Dystrophy type 1, Small molecule, Recombinant protein production, Orphan disease, Drug Design, RNA targeting, Patient-derived myoblasts, Organic Synthesis, TR-FRET, ncRNA, Ciències naturals, químiques, físiques i matemàtiques

وصف الملف: application/pdf

الوصول الحر: http://hdl.handle.net/10803/687314Test

المؤلفون: COHEN, ALAN B. abcohen@bu.edu

المصدر: Milbank Quarterly. Mar2024, Vol. 102 Issue 1, p5-10. 6p.

مصطلحات موضوعية: *QUALITY assurance, ORPHAN drug laws, SERIAL publications, HEALTH policy, SPECIAL days, PEOPLE with disabilities

مصطلحات جغرافية: UNITED States

المؤلفون: Lexchin, Joel^1,2 (AUTHOR) jlexchin@yorku.ca

المصدر: JGIM: Journal of General Internal Medicine. May2024, Vol. 39 Issue 7, p1276-1279. 4p.

مصطلحات موضوعية: *ORPHAN drugs, *DRUG approval, *CROSS-sectional method, *BIOMARKERS

الشركة/الكيان: CANADA. Health Canada

المؤلفون: Hu, Yubo¹ (AUTHOR) huyubo202209@163.com, Pang, Zhiqiang¹ (AUTHOR)

المصدر: PLoS ONE. 5/6/2024, Vol. 19 Issue 5, p1-37. 37p.

مصطلحات موضوعية: *STATISTICAL correlation, *ORPHAN drugs, *GROUP decision making, *CLINICAL medicine, *FUZZY sets, *MACHINE translating

المؤلفون: Orimoloye, Helen T, Nguyen, Nicholas, Deng, Chuanjie, Saechao, Chai, Ritz, Beate, Olsen, Jorn, Hansen, Johnni, Heck, Julia E

مصطلحات موضوعية: Epidemiology, Biomedical and Clinical Sciences, Health Sciences, Oncology and Carcinogenesis, Orphan Drug, Arthritis, Clinical Research, Brain Disorders, Hematology, Neurosciences, Pediatric Cancer, Cancer, Autoimmune Disease, Childhood Leukemia, Pediatric, Brain Cancer, Rare Diseases, Pediatric Research Initiative, 2.1 Biological and endogenous factors, Aetiology, Inflammatory and immune system, Reproductive health and childbirth, Good Health and Well Being, Maternal autoimmune diseases, childhood cancer, inflammatory bowel disease, leukemia, pregnancy, psoriasis, rheumatoid arthritis

وصف الملف: application/pdf

الوصول الحر: https://escholarship.org/uc/item/3xz469s1Test
https://escholarship.org/content/qt3xz469s1/qt3xz469s1.pdfTest

المؤلفون: Rivas, Victor N, Crofton, Amanda E, Jauregui, Carina E, Wouters, Jalena R, Yang, Betty S, Wittenburg, Luke A, Kaplan, Joanna L, Hwee, Darren T, Murphy, Anne N, Morgan, Bradley P, Malik, Fady I, Harris, Samantha P, Stern, Joshua A

المصدر: Scientific Reports. 14(1)

مصطلحات موضوعية: Veterinary Sciences, Agricultural, Veterinary and Food Sciences, Biomedical and Clinical Sciences, Heart Disease, Cardiovascular, Pediatric, Pediatric Cardiomyopathy, Clinical Trials and Supportive Activities, Orphan Drug, Clinical Research, Rare Diseases, 5.1 Pharmaceuticals, Evaluation of treatments and therapeutic interventions, Development of treatments and therapeutic interventions, 6.1 Pharmaceuticals, Animals, Cats, Cardiomyopathy, Hypertrophic, Cardiac Myosins, Cat Diseases, Male, Female, Ventricular Outflow Obstruction, Systole, Echocardiography, Cross-Over Studies, Cat, Left ventricular outflow tract obstruction, Myosin-inhibitor, Obstructive hypertrophic cardiomyopathy, Pharmacodynamics

وصف الملف: application/pdf

الوصول الحر: https://escholarship.org/uc/item/4db1b6gvTest
https://escholarship.org/content/qt4db1b6gv/qt4db1b6gv.pdfTest

المؤلفون: Cicenas, Jonas^1,2 (AUTHOR) j.cicenas@mapkinases.eu, Simkus, Jokubas^1,3 (AUTHOR)

المصدر: Cancers. Apr2024, Vol. 16 Issue 8, p1555. 6p.

مصطلحات موضوعية: *ORPHAN drugs, *HEMATOLOGIC malignancies, *BREAST tumors, *DRUG approval, *METASTASIS, *MOLECULAR structure, *CYCLIN-dependent kinases, *GENOMES, *CHEMICAL inhibitors

الشركة/الكيان: UNITED States. Food & Drug Administration

المؤلفون: Kostadinov, Kostadin¹ (AUTHOR) ivelina.popova@mu-plovdiv.bg, Popova-Sotirova, Ivelina¹ (AUTHOR) yuliyana.marinova@mu-plovdiv.bg, Marinova, Yuliyana¹ (AUTHOR) nina.musurlieva@mu-plovdiv.bg, Musurlieva, Nina¹ (AUTHOR) georgi.iskrov@mu-plovdiv.bg, Iskrov, Georgi^1,2 (AUTHOR) rumen.stefanov@mu-plovdiv.bg, Stefanov, Rumen^1,2 (AUTHOR)

المصدر: Cancers. Apr2024, Vol. 16 Issue 8, p1489. 19p.

مصطلحات موضوعية: *HEALTH services accessibility, *ORPHAN drugs, *HEALTH insurance reimbursement, *RARE diseases, *HEALTH policy, *GOVERNMENT agencies, *RETROSPECTIVE studies, *DESCRIPTIVE statistics, *MANN Whitney U Test, *MEDICAL records, *ACQUISITION of data, *TUMORS, *HEALTH equity, *DATA analysis software, *MEDICAL care costs

مصطلحات جغرافية: BULGARIA