Search Results - "rare diseases"

Safety, efficacy and pharmacokinetics of palivizumab in off-label neonates, infants, and young children at risk for serious respiratory syncytial virus infection: a multicenter pha... by Masaaki Mori, Kanako Yoshizaki, Shinichi Watabe, Mika Ishige, Akinari Hinoki, Takuya Kondo, Tomoaki Taguchi, Hisaya Hasegawa, Tomoko Hatata, Naoyuki Tanuma, Kosuke Kirino, Akihiro Hirakawa, Takuya Naruto, Minoru Imai, Ryuji Koike, Kenichiro Hosoi, Satoshi Kusuda

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A new genetic variant of hereditary apolipoprotein A-I amyloidosis: a case-report followed by discussion of diagnostic challenges and therapeutic options by Myrto Moutafi, Dimitrios C. Ziogas, Spyros Michopoulos, Tina Bagratuni, Vassiliki Vasileiou, Laura Verga, Giampaolo Merlini, Giovanni Palladini, Charis Matsouka, Meletios A. Dimopoulos, Efstathios Kastritis

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Reimbursement of Orphan Drugs in Europe in Relation to the Type of Authorization by the European Medicines Agency and the Decision Making Based on Health Technology Assessment by Krzysztof Piotr Malinowski, Paweł Kawalec, Wojciech Trabka, Christoph Sowada, Andrzej Pilc, Andrzej Pilc

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Hematuria in a Patient with Non-malignant Bladder Nodules by Terese S Bergheim, Visal Nga, Andrew C Burg, Bahman Chavoshan

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Acute intestinal obstruction in systemic lupus Erythematosus: Case report and review of the literature by Bouali Mounir, Eddaoudi Yassine, El Wassi Anas, El Bakouri Abdelilah, El Hattabi Khalid, Bensardi Fatimazahra, Fadil Abdelaziz

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Efficacy and safety of lumasiran for infants and young children with primary hyperoxaluria type 1: 30-month analysis of the phase 3 ILLUMINATE-B trial by Yaacov Frishberg, Wesley Hayes, Hadas Shasha-Lavsky, Hadas Shasha-Lavsky, David J. Sas, Mini Michael, Anne-Laure S, Julien Hogan, Richard Willey, John M. Gansner, Daniella Magen

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Consequências da judicialização das políticas de saúde: custos de medicamentos para as mucopolissacaridoses Consequences of the judicialization of health policies: the cost of... by Debora Diniz, Marcelo Medeiros, Ida Vanessa D. Schwartz

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Methodological and regulatory aspects of pharmaceutical development of biological products by D. V. Kurkin, E. I. Morkovin, D. A. Bakulin, A. V. Zaborovsky, I. E. Makarenko, R. V. Drai, A. G. Solodovnikov, V. I. Petrov, K. N. Koryanova, N. A. Lycheva, S. A. Voskresensky, A. V. Strygin, Yu. A. Kolosov, Yu. V. Gorbunova, O. V. Ivanova

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Prehospital emergency medicine for children receiving palliative home care in Germany-a cross-sectional, exploratory study of EMS providers by Holger Hauch, Holger Hauch, Naual El Mohaui, Vera Vaillant, Michael Sander, Peter Kriwy, Marius Rohde, Johannes Wolff, Daniel Berthold, Emmanuel Schneck

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Dental and maxillomandibular incidental findings in panoramic radiography among individuals with mucopolysaccharidosis: a cross-sectional study by Natália Cristina Ruy CARNEIRO, Lucas Guimarães ABREU, Roselaine Moreira Coelho MILAGRES, Tania Mara Pimenta AMARAL, Carlos FLORES-MIR, Isabela Almeida PORDEUS, Ana Cristina BORGES-OLIVEIRA

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Síndrome de Erasmus Associada à Policondrite Recidivante: Relato de Caso / Erasmus' Syndrome in Association with Relapsing Polychondritis: a Case Report by Jacqueline Foelkel Pignatari, Marcela Maria da Silva, Maria Zilda Cardoso, Clarissa Santos de Carvalho Ribeiro

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Medical crowdfunding in a healthcare system with universal coverage: an exploratory study by Ágnes Lublóy

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Coenzyme q10 liquid supplementation in dyslipidemic subjects with statin-related clinical symptoms: a double-blind, randomized, placebo-controlled study by Derosa G, D'Angelo A, Maffioli P

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Expiry of veterinary medicines in supply outlets in Central Uganda: prevalence, management and associated factors by Stevens Kisaka, Frank K. Tumwebaze

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Novel CAD gene mutations in a boy with developmental and epileptic encephalopathy 50 with dramatic response to uridine therapy: a case report and a review of the literature by Lifen Duan, Lei Ye, Runxiu Yin, Ying Sun, Wei Yu, Yi Zhang, Haiyan Zhong, Xinhua Bao, Xin Tian

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Clinical and non-clinical aspects of reimbursement policy for orphan drugs in selected European countries by Szczepan Jakubowski, Przemysław Holko, Rafał Nowak, Marisa Warmuth, Marc Dooms, Outi Salminen, Lucas Cortial, Gisbert W. Selke, Christina Georgi, Einar Magnússon, Salvatore Crisafulli, Fons Strijbosch, Tanja Mueller, Eleanor Grieve, Immaculada Danés, Paweł Kawalec

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Cardiopulmonary resuscitation in pediatric patients under palliative home care - A multicenter retrospective study by Emmanuel Schneck, Gisela Janßen, Vera Vaillant, Thomas Voelker, Oliver Dechert, Laura Trocan, Lioba Schmitz, Marius Rohde, Michael Sander, Holger Hauch

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Whole genome sequencing diagnostic yield for paediatric patients with suspected genetic disorders: systematic review, meta-analysis, and GRADE assessment by Mario Cesare Nurchis, Gerardo Altamura, Maria Teresa Riccardi, Francesca Clementina Radio, Giovanni Chillemi, Enrico Silvio Bertini, Jacopo Garlasco, Marco Tartaglia, Bruno Dallapiccola, Gianfranco Damiani

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Dental anomalies in individuals with osteogenesis imperfecta: a systematic review and meta-analysis of prevalence and comparative studies by Heloisa Vieira PRADO, Enio Cássio Barreto SOARES, Natália Cristina Ruy CARNEIRO, Ivanete Cláudia de Oliveira VILAR, Lucas Guimarães ABREU, Ana Cristina BORGES-OLIVEIRA

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Real-world data: a comprehensive literature review on the barriers, challenges, and opportunities associated with their inclusion in the health technology assessment process by Konstantinos Zisis, Konstantinos Zisis, Elpida Pavi, Mary Geitona, Kostas Athanasakis

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