المؤلفون: Piette, Y, Van den Bossche, F, Aerts, J, Aerts, N, Ajeganova, S, Badot, V, Berghen, N, Blockmans, D, Brusselle, G, Caeyers, N, De Decker, M, De Haes, P, De Cock, C, De Keyser, F, De Langhe, E, Delcroix, M, De Nutte, H, De Pauw, M, Depicker, A, De Sutter, A, De Sutter, J, Du Four, T, Frank, C, Goubau, J, Guiot, Julien, Gutermuth, J, Heeman, L, Houssiau, F, Hennes, I, Lenaerts, J, Lintermans, A, Loeys, B, Luyten, H, Maeyaert, B, Malfait, F, Moeyersoons, A, Mostmans, Y, Nijs, J, Poppe, B, Polfliet, K, Ruttens, D, Sabato, V, Schoeters, E, Slabbynck, H, Stuer, A, Tamirou, F, Thevissen, Kristof, Van Kersschaever, G, Vanneuville, B, Van Offel, J, Vanthuyne, M, Van Wabeke, J, Verbist, C, Vos, I, Westhovens, R, Wuyts, W, Yserbyt, J, Smith, V

المصدر: Acta Clinica Belgica, 79 (1), 26 - 33 (2024-02)

مصطلحات موضوعية: European reference networks, Flemish Network of rare diseases, patient pathways, rare connective tissue diseases, systemic sclerosis, Humans, Rare Diseases/complications, Rare Diseases/epidemiology, Rare Diseases/therapy, Scleroderma, Systemic/diagnosis, Scleroderma, Systemic/therapy, Connective Tissue Diseases/diagnosis, Connective Tissue Diseases/complications, Lung Diseases, Interstitial/diagnosis, Lung Diseases, Interstitial/therapy, Lung Diseases, Interstitial/complications, Scleroderma, Diffuse, General Medicine, Human health sciences, Cardiovascular & respiratory systems, Rheumatology, Sciences de la santé humaine, Systèmes cardiovasculaire & respiratoire, Rhumatologie

Relation: https://www.tandfonline.com/doi/pdf/10.1080/17843286.2023.2280737; urn:issn:1784-3286; urn:issn:2295-3337

URL الوصول: https://orbi.uliege.be/handle/2268/311069

المساهمون: Parvin, Simon D., Earnshaw, J. J. (Jonothan J.), Joint Vascular Research Group.

مصطلحات موضوعية: Blood-vessels -- Diseases., Vascular Diseases., Vascular Diseases -- diagnosis., Vascular Diseases -- therapy., Rare Diseases -- diagnosis., Rare Diseases -- therapy.

المؤلفون: Vieira, JP

مصطلحات موضوعية: Orphan Drug Production, HDE NEU PED, Rare Diseases/therapy

URL الوصول: https://explore.openaire.eu/search/publication?articleId=od______2340::62e3a4e8399ef7439c15863ceacc6921
https://hdl.handle.net/10400.17/4242

المؤلفون: Fulvio Mavilio

المساهمون: Approches génétiques intégrées et nouvelles thérapies pour les maladies rares (INTEGRARE), École pratique des hautes études (EPHE), Université Paris sciences et lettres (PSL)-Université Paris sciences et lettres (PSL)-Université d'Évry-Val-d'Essonne (UEVE)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Généthon, École Pratique des Hautes Études (EPHE)

المصدر: Gene Ther
Gene Ther, 2017, 24 (9), pp.590-592. ⟨10.1038/gt.2017.36⟩

مصطلحات موضوعية: 0301 basic medicine, Biomedical Research, Drug Industry, Rare Diseases/*therapy, [SDV]Life Sciences [q-bio], Academic Medical Centers/*organization &, Gene transfer, Legislation, Genetic Therapy/*methods, Drug Industry/*organization &, administration, Genetic therapy, Orphan drug, 03 medical and health sciences, 0302 clinical medicine, Rare Diseases, Genetics, Medicine, Humans, Molecular Biology, Drug industry, Intersectoral Collaboration, health care economics and organizations, Academic Medical Centers, business.industry, Genetic Therapy, Public relations, 3. Good health, Biotechnology, 030104 developmental biology, 030220 oncology & carcinogenesis, Molecular Medicine, Biomedical Research/methods/organization &, business

URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::56750c9b10d18a5d0fdad2de5a545d40
https://hal.archives-ouvertes.fr/hal-02880997

المؤلفون: Carlo Tomino, Penilla Gunther, Josep Torrent-Farnell, Sheela Upadhyaya, Elena Nicod, Jean Louis Roux, Yann Le Cam, Michael Schlander, Adam Hutchings, Karen Facey, David Taylor, Lieven Annemans, Ségolène Aymé, Lugdivine Le Dez, Michele Reni

المساهمون: BMC, BMC, Department of Public Health [Ghent, Belgium], Institut du Cerveau et de la Moëlle Epinière = Brain and Spine Institute (ICM), Université Pierre et Marie Curie - Paris 6 (UPMC)-Institut National de la Santé et de la Recherche Médicale (INSERM)-CHU Pitié-Salpêtrière [AP-HP], Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Sorbonne Université (SU)-Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Sorbonne Université (SU)-Centre National de la Recherche Scientifique (CNRS), EURORDIS-Rare Diseases Europe (Bureau de Paris), EURORDIS - Plateforme Maladies Rares [Paris], Institute of Population Health Sciences and Informatics [Edinburgh, Scotland, U.K.], University of Edinburgh, The Swedish Delegation to the Nordic Council [Stockholm, Sweden], Swedish Parliament [Stockholm, Sweden], Centre for Research on Health and Social Care Management [Milan, Italy] (CERGAS), Bocconi University [Milan, Italy], Ospedale San Raffaele Scientific Institute [Milan, Italy], Fondazione IRCCS Instituto Nazionale dei Tumori [Milan, Italy], Health Economics [Heidelberg, Germany], Heidelberg University, Division of Health Economics [Heidelberg, Germany], German Cancer Research Center - Deutsches Krebsforschungszentrum [Heidelberg] (DKFZ), Institute for Innovation & Valuation in Health Care [Wiesbaden, Germany], Pharmaceutical and Public Health Policy [London, UK], University College of London [London] (UCL), Clinical Research [Rome, Italy], Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS San Raffaele Pisana), Clinical Pharmacology and Therapeutic [Barcelona, Spain], Universitat Autònoma de Barcelona (UAB), National Institute for Health and Care Excellence [Manchester, UK] (NICE), Dolon Ltd [London, UK], European government relations and public policy at Celgene [Brussels, Belgium], EUCOPE and Celgene Corporation., Universiteit Gent = Ghent University [Belgium] (UGENT), Sorbonne Université (SU)-Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Sorbonne Université (SU)-Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Centre National de la Recherche Scientifique (CNRS)

المصدر: Orphanet Journal of Rare Diseases
Orphanet Journal of Rare Diseases, 2016, 12 (1), pp.50. ⟨10.1186/s13023-017-0601-9⟩
Orphanet Journal of Rare Diseases, BioMed Central, 2016, 12 (1), pp.50. ⟨10.1186/s13023-017-0601-9⟩
Annemans, L, Aymé, S, Le Cam, Y, Facey, K, Gunther, P, Nicod, E, Reni, M, Roux, J-L, Schlander, M, Taylor, D, Tomino, C, Torrent-Farnell, J, Upadhyaya, S, Hutchings, A & Le Dez, L 2017, ' Recommendations from the European Working Group for Value Assessment and Funding Processes in Rare Diseases (ORPH-VAL) ', Orphanet journal of rare diseases, vol. 12, no. 1, pp. 50 . https://doi.org/10.1186/s13023-017-0601-9

مصطلحات موضوعية: Value (ethics), medicine.medical_specialty, Orphan Drug Production, Pricing and reimbursement, Universal design, Orphan Drug Production/economics, [SDV]Life Sciences [q-bio], Guidelines, Drug Costs, 03 medical and health sciences, 0302 clinical medicine, Consistency (negotiation), Rare Diseases, Value assessment, medicine, Humans, Genetics(clinical), Pharmacology (medical), 030212 general & internal medicine, Health technology assessment, Position Statement, Genetics (clinical), Reimbursement, Health policy, Medicine(all), Orphan medicinal products, business.industry, 030503 health policy & services, Public health, Health Policy, Environmental resource management, General Medicine, Public relations, Multiple-criteria decision analysis, 3. Good health, [SDV] Life Sciences [q-bio], Europe, Sustainability, Business, 0305 other medical science, Rare Diseases/therapy

وصف الملف: application/pdf

URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::91b6eb4fa44222d5d48436582b9cdcf0
https://pubmed.ncbi.nlm.nih.gov/28283046

المؤلفون: Volpi, S., Picco, P., Caorsi, R., Candotti, F., Gattorno, M.

المصدر: Pediatric rheumatology online journal, vol. 14, no. 1, pp. 35

مصطلحات موضوعية: Aortic Diseases/genetics, Aortic Diseases/immunology, Arthritis, Juvenile/diagnosis, Arthritis, Juvenile/immunology, Autoimmune Diseases/diagnosis, Autoimmune Diseases/genetics, Autoimmune Diseases/immunology, Autoimmune Diseases/therapy, Autoimmune Diseases of the Nervous System/diagnosis, Autoimmune Diseases of the Nervous System/immunology, Dental Enamel Hypoplasia/genetics, Dental Enamel Hypoplasia/immunology, Homozygote, Humans, Interferon Type I/genetics, Interferon Type I/immunology, Lupus Erythematosus, Systemic/diagnosis, Lupus Erythematosus, Systemic/genetics, Lupus Erythematosus, Systemic/immunology, Metacarpus/abnormalities, Metacarpus/immunology, Muscular Diseases/genetics, Muscular Diseases/immunology, Mutation/genetics, Mutation/immunology, Nervous System Malformations/diagnosis, Nervous System Malformations/immunology, Odontodysplasia/genetics, Odontodysplasia/immunology, Osteochondrodysplasias/genetics, Osteochondrodysplasias/immunology, Osteoporosis/genetics, Osteoporosis/immunology, Proteome/genetics, Proteome/immunology, Rare Diseases/diagnosis, Rare Diseases/immunology, Rare Diseases/therapy, Signal Transduction, Vascular Calcification/genetics, Vascular Calcification/immunology

وصف الملف: application/pdf

URL الوصول: https://explore.openaire.eu/search/publication?articleId=od______1900::6259d649ae88c66ae8976fb1d7306c11
https://serval.unil.ch/notice/serval:BIB_F53B15A22E70

المؤلفون: Constance Barazzone, Argiroffo, A, Superti-Furga

المصدر: Revue médicale suisse, Vol. 8, No 329 (2012) pp. 395-6

مصطلحات موضوعية: Rare Diseases, ddc:618, Biomedical Research, Humans, Child, Rare Diseases/therapy

URL الوصول: https://explore.openaire.eu/search/publication?articleId=pmid_dedup__::a58e11ece1506ee660418bbbc4d148dd
https://archive-ouverte.unige.ch/unige:31214

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المصدر: Bulletin du cancer, 104 (12S

مصطلحات الفهرس: Sciences bio-médicales et agricoles, Allografts, Autografts, France, Hematopoietic Stem Cell Transplantation -- standards, Hodgkin Disease -- therapy, Humans, Lymphoma, Mantle-Cell -- therapy, Lymphoma, Non-Hodgkin -- therapy, Rare Diseases -- therapy, Recurrence, Retreatment -- standards, Societies, Medical, Allogeneic stem cell transplantation, Allogreffe, Guidelines, Lymphoma, Lymphome, Recommandations, info:eu-repo/semantics/article, info:ulb-repo/semantics/articlePeerReview, info:ulb-repo/semantics/openurl/article

URL: https://dipot.ulb.ac.be/dspace/bitstream/2013/266733/1/Elsevier_250360.pdf
http://hdl.handle.net/2013/ULB-DIPOT:oai:dipot.ulb.ac.be:2013/266733
http://worldcat.org/search?q=on:EQY+http://difusion-oai.ulb.ac.be/oai/request+DCG_ENTIRE_REPOSITORY+CNTCOLL