Works matching DE "TREATMENT of rare diseases"
Results: 298
Surgical treatment of sacroiliac joint infection.
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- Journal of Orthopaedics & Traumatology, 2013, v. 14, n. 2, p. 121, doi. 10.1007/s10195-013-0233-3
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- Article
Mitochondrial disorders as windows into an ancient organelle.
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- Nature, 2012, v. 491, n. 7424, p. 374, doi. 10.1038/nature11707
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- Article
Perspective: Not just for women.
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- Nature, 2012, v. 485, n. 7400, p. S66, doi. 10.1038/485S66a
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- Article
Seven days: 1?7 April 2011.
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- Nature, 2011, v. 472, n. 7341, p. 10, doi. 10.1038/472010a
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- Article
Cells snag top modelling job.
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- Nature, 2011, v. 469, n. 7330, p. 279, doi. 10.1038/469279a
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- Article
Are Corticosteroids the Mainstay of Treatment for Multisystem Inflammatory Syndrome in Children?
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- Central European Journal of Paediatrics, 2021, v. 17, n. 2, p. 103, doi. 10.5457/p2005-114.295
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- Article
Ethical challenges and opportunities in the development and approval of novel therapeutics for rare diseases.
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- Medicine Access @ Point of Care, 2023, v. 7, p. 1, doi. 10.1177/27550834231177507
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- Article
Large-scale computational drug repositioning to find treatments for rare diseases.
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- NPJ Systems Biology & Applications, 2018, v. 4, n. 1, p. N.PAG, doi. 10.1038/s41540-018-0050-7
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- Article
Good-quality research in rare diseases: trials and tribulations.
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- Pediatric Nephrology, 2016, v. 31, n. 11, p. 2017, doi. 10.1007/s00467-016-3323-7
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- Article
Rare Disease Funding: A Rawlsian Approach to Orphan Drug Legislation.
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- Penn Bioethics Journal, 2015, v. 11, n. 2, p. 13
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Letter from the Editor.
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- Penn Bioethics Journal, 2015, v. 11, n. 2, p. 4
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A Rare Case of Cutaneous Leishmaniasis Presenting as Rhinophyma.
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- Iranian Journal of Pathology, 2015, v. 10, n. 2, p. 155
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Examining whether the information-motivation-behavioral skills model predicts medication adherence for patients with a rare disease.
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- Patient Preference & Adherence, 2017, v. 11, p. 75, doi. 10.2147/PPA.S115272
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- Article
Towards government-funded special biomedical research programs to combat rare diseases in China.
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- BioScience Trends, 2015, v. 9, n. 2, p. 88, doi. 10.5582/bst.2015.01048
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- Article
"It is like a jungle gym, and everything is under construction": The parent's perspective of caring for a child with a rare disease.
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- Child: Care, Health & Development, 2019, v. 45, n. 1, p. 96, doi. 10.1111/cch.12628
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- Article
'It's a privilege to look after these children'.
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- Nursing Standard, 2017, v. 31, n. 34, p. 18, doi. 10.7748/ns.31.34.18.s22
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- Article
Outcome measures of disease activity for rare autoimmune rheumatic diseases.
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- British Journal of Hospital Medicine (17508460), 2018, v. 79, n. 7, p. 396, doi. 10.12968/hmed.2018.79.7.396
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- Article
Rarely mentioned: how we arrived at the quantitative definition of a rare disease.
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- Baylor University Medical Center Proceedings, 2022, v. 35, n. 4, p. 498, doi. 10.1080/08998280.2022.2048613
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- Article
Clinical experience with lanreotide for the treatment of acromegaly.
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- Expert Review of Endocrinology & Metabolism, 2012, v. 7, n. 2, p. 139, doi. 10.1586/EEM.11.93
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On the use of propensity scores in case of rare exposure.
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- 2016
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- journal article
The European Rare Diseases Therapeutic Initiative.
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- PLoS Medicine, 2005, v. 2, n. 9, p. 845, doi. 10.1371/journal.pmed.0020243
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LUMBAR INTRAMEDULLARY DERMOID TUMOR IN AN ADULT: A CASE REPORT.
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- 2021
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- Case Study
Correction to: Treatment of Rare and Aggressive Pancreatic Carcinosarcoma.
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- ACG Case Reports Journal, 2020, v. 7, n. 8, p. 1, doi. 10.14309/crj.0000000000000435
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- Article
The unintended effect of the Orphan Drug Act on the adoption of open innovation.
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- Science & Public Policy (SPP), 2016, v. 43, n. 6, p. 840, doi. 10.1093/scipol/scw001
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- Article
Pathway Maps of Orphan and Complex Diseases Using an Integrative Computational Approach.
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- BioMed Research International, 2020, p. 1, doi. 10.1155/2020/4280467
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- Article
Chronic neutrophilic leukemia, an extremely rare cause of neutrophilia in childhood: Cure with hematopoietic stem cell transplantation.
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- Pediatric Transplantation, 2018, v. 22, n. 5, p. 1, doi. 10.1111/petr.13199
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- Article
Orphan drug development is not taking off.
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- British Journal of Clinical Pharmacology, 2009, v. 67, n. 5, p. 494, doi. 10.1111/j.1365-2125.2009.03369.x
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- Article
Optimizing medical trips to care for those with rare genetic diseases in remote settings: lessons learned from xeroderma pigmentosum.
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- International Journal of Dermatology, 2017, v. 56, n. 5, p. e104, doi. 10.1111/ijd.13586
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- Article
Interaction orthodontie-implantologie et prothèse dans les maladies rares : l'exemple des oligodonties.
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- Revue d'Orthopédie Dento-Faciale, 2014, v. 48, n. 1, p. 55, doi. 10.1051/odf/2013506
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- Article
Pathologies rares de la face et de la cavité buccale : spécificités de la collaboration ortho-pédo.
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- Revue d'Orthopédie Dento-Faciale, 2014, v. 48, n. 1, p. 37, doi. 10.1051/odf/2013508
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The Journey to Ancient Ithaca: addressing the diagnostic odyssey of rare disease through systems-level interventions.
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- University of Toronto Medical Journal, 2020, v. 97, n. 3, p. 37
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- Article
A rare case of pilonidal sinus of the umbilicus.
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- Central European Journal of Urology, 2009, v. 62, n. 2, p. 116, doi. 10.5173/ceju.2009.02.art15
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- Article
Entanglements of rare diseases in the Baltic Sea region.
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- 2025
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- Book Review
INDUSTRY PIPELINE.
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- Ocular Surgery News, 2024, v. 42, n. 8, p. 24
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- Article
Clinical trials for rare diseases need new, specific methodological approaches.
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- Ocular Surgery News, 2024, v. 42, n. 3, p. 13
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INDUSTRY NEWS IN BRIEF.
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- Ocular Surgery News, 2017, v. 35, n. 22, p. 19
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- Article
Management of rare diseases of the Head, Neck and Teeth: results of a French population-based prospective 8-year study.
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- 2017
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- journal article
Determinants of orphan drugs prices in France: a regression analysis.
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- 2017
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- journal article
Orphan medicinal products in Europe and United States to cover needs of patients with rare diseases: an increased common effort is to be foreseen.
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- 2017
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- journal article
Review of 11 national policies for rare diseases in the context of key patient needs.
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- 2017
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- journal article
Off-label use of orphan medicinal products: a Belgian qualitative study.
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- Orphanet Journal of Rare Diseases, 2016, v. 11, p. 1, doi. 10.1186/s13023-016-0507-y
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- Article
Combined integrated protocol/basket trial design for a first-in-human trial.
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- Orphanet Journal of Rare Diseases, 2016, v. 11, p. 1, doi. 10.1186/s13023-016-0494-z
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State of rare disease management in Southeast Asia.
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- 2016
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Systematic review on the evaluation criteria of orphan medicines in Central and Eastern European countries.
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- 2016
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Directions for new developments on statistical design and analysis of small population group trials.
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- 2016
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- journal article
Rare disease landscape in Brazil: report of a successful experience in inborn errors of metabolism.
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- 2016
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- journal article
Catastrophic expenditure and impoverishment of patients affected by 7 rare diseases in China.
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- 2016
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- journal article
Pharmaceutical expenditure on drugs for rare diseases in Canada: a historical (2007-13) and prospective (2014-18) MIDAS sales data analysis.
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- 2016
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- journal article
The availability and affordability of orphan drugs for rare diseases in China.
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- 2016
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- journal article
Principles for consistent value assessment and sustainable funding of orphan drugs in Europe.
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- Orphanet Journal of Rare Diseases, 2015, v. 10, n. 1, p. 1, doi. 10.1186/s13023-015-0269-y
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- Article