Orphan diseases — rare but important!

by article Editorial
Published 2019-06-01

Orphan diseases — rare but important!

by article Editorial
Published 2019-06-01

NORD. A beacon of hope for people with rare disorders

by Stefanie Putkowski
Published 2010-04-01

Building a trusted framework for uncertainty assessment in rare diseases: suggestions for improvement (Response to “TRUST4RD: tool for reducing uncertainties in the evidence genera...

by Sabine E. Grimm, Xavier Pouwels, Bram L. T. Ramaekers, Ben Wijnen, Saskia Knies, Janneke Grutters, Manuela A. Joore
Published 2021-02-01

Orphan Disease in Structure of Recurrent Lower Respiratory Tract Diseases in Children from One To Five

by Zubarenko O. V., Koval Larisa, Doykova Katherina, Kopiyka Ganna, Skrypnyk V. V., Lotysh Nadiya, Papinko R. M.
Published 2017-05-01

ASSESSMENT OF NECESSARY FINANCIAL EXPENSES ON THE DRUG SUPPLY OF THE PATIENTS WITH ORPHAN DISEASES IN THE RUSSIAN FEDERATION

by M. V. Sura, K. V. Gerasimova, V. V. Omelyanovskiy, M. V. Avksentyeva
Published 2015-03-01

Primary ciliary dyskinesia: modern approaches to the diagnostics and treatment

by A. E. Bogorad, S. E. Diakova, Yu. L. Mizernitsky
Published 2019-11-01

Johanson—Blizzard syndrome

by L. S. Baleva, Yu. M. Kagan, L. I. Danilycheva, S. F. Bluth
Published 2016-03-01

Disease awareness or subtle product placement? Orphan diseases featured in the television series “House, M.D.” - a cross-sectional analysis

by Konstantin Mechler, Juliane Rausch, William K. Mountford, Markus Ries
Published 2020-03-01

Morphological verification of renal lesion as a compulsory stage in the diagnosis of Fabry disease

by P V Glybochko, N A Mukhin, A A Svistunov, V V Fomin, M Iu Brovko, V A Varshavskiĭ, S V Moiseev, E M Shilov, V V Panasiuk
Published 2014-12-01

Barriers for Access to New Medicines: Searching for the Balance Between Rising Costs and Limited Budgets

by Brian Godman, Brian Godman, Brian Godman, Brian Godman, Anna Bucsics, Patricia Vella Bonanno, Wija Oortwijn, Wija Oortwijn, Celia C. Rothe, Alessandra Ferrario, Simone Bosselli, Andrew Hill, Antony P. Martin, Antony P. Martin, Steven Simoens, Amanj Kurdi, Amanj Kurdi, Mohamed Gad, Jolanta Gulbinovič, Angela Timoney, Angela Timoney, Tomasz Bochenek, Ahmed Salem, Iris Hoxha, Robert Sauermann, Amos Massele, Augusto Alfonso Guerra, Augusto Alfonso Guerra, Guenka Petrova, Zornitsa Mitkova, Gnosia Achniotou, Ott Laius, Catherine Sermet, Gisbert Selke, Vasileios Kourafalos, John Yfantopoulos, Einar Magnusson, Roberta Joppi, Margaret Oluka, Hye-Young Kwon, Arianit Jakupi, Francis Kalemeera, Joseph O. Fadare, Oyvind Melien, Maciej Pomorski, Magdalene Wladysiuk, Vanda Marković-Peković, Vanda Marković-Peković, Ileana Mardare, Dmitry Meshkov, Tanja Novakovic, Jurij Fürst, Dominik Tomek, Corrine Zara, Eduardo Diogene, Johanna C. Meyer, Rickard Malmström, Björn Wettermark, Björn Wettermark, Zinhle Matsebula, Stephen Campbell, Stephen Campbell, Alan Haycox
Published 2018-12-01

Rare Genetic Disorders Affecting the Periodontal Supporting Tissues in Adolescence

by Ines Kapferer-Seebacher, Lena Foradori, Johannes Zschocke, Reinhard Schilke
Published 2021-07-01

EPILEPSY IN CHILDREN WITH MUCOPOLYSACCHARIDOSIS: EPIDEMIOLOGY, CLINICAL AND ELECTROENCEPHALOGRAPHIC FEATURES

by Victoriya A. Aysina, Aleksandr A. Buksh, Ludmila M. Kuzenkova, Olga V. Kozhevnikova, Vladislav V. Lebedev, Andrey A. Gazaryan, Tatyana V. Podkletnova, Sofiya G. Popovich
Published 2018-07-01

A systematic review of moral reasons on orphan drug reimbursement

by Bettina M. Zimmermann, Johanna Eichinger, Matthias R. Baumgartner
Published 2021-06-01

DRUG FLURENISID AS AN IMMUNOMODULATOR FOR THE TREATMENT OF ORPHAN DISEASES

by Мaria Kovalenko
Published 2017-12-01

Late Diagnosis of Infants with PCD and Neonatal Respiratory Distress

by Myrofora Goutaki, Florian S. Halbeisen, Angelo Barbato, Suzanne Crowley, Amanda Harris, Robert A. Hirst, Bülent Karadag, Vendula Martinu, Lucy Morgan, Christopher O’Callaghan, Ugur Ozçelik, Sergio Scigliano, Santiago Ucros, Panayiotis Yiallouros, Sven M. Schulzke, Claudia E. Kuehni
Published 2020-09-01

The Value of Mouse Models of Rare Diseases: A Spanish Experience

by Silvia Murillo-Cuesta, Silvia Murillo-Cuesta, Silvia Murillo-Cuesta, Rafael Artuch, Rafael Artuch, Fernando Asensio, Pedro de la Villa, Mara Dierssen, Mara Dierssen, Mara Dierssen, Jose Antonio Enríquez, Jose Antonio Enríquez, Cristina Fillat, Cristina Fillat, Stéphane Fourcade, Stéphane Fourcade, Borja Ibáñez, Borja Ibáñez, Borja Ibáñez, Lluis Montoliu, Lluis Montoliu, Eduardo Oliver, Eduardo Oliver, Aurora Pujol, Aurora Pujol, Aurora Pujol, Eduardo Salido, Eduardo Salido, Mario Vallejo, Mario Vallejo, Isabel Varela-Nieto, Isabel Varela-Nieto, Isabel Varela-Nieto
Published 2020-10-01

The paediatric rheumatologist and orphan disease – a story without happy ending

by Justyna Roszkiewicz, Małgorzata Biernacka-Zielińska, Elżbieta Smolewska
Published 2016-07-01

Use of Implantable Venous Port Systems in the Treatment of Children with Orphan Diseases (Mucopolysaccharidosis and Pompe Disease): Case Series

by M. Yu. Rykov, I. V. Filinov, E. I. Petrov, N. D. Vashakmadze, A. K. Gevorkyan, E. N. Arkhipova, I. V. Sil’nova, E. N. Basargina, N. V. Buchinskaya, A. I. Ivanov, E. A. Isupova, M. M. Kostik, N. A. Abramova, O. V. Kalashnikova, V. G. Chasnyk, A. E. Aleksandrov, D. A. Morozov, V. G. Polyakov
Published 2015-09-01

Think Big, Start Small: How Nanomedicine Could Alleviate the Burden of Rare CNS Diseases

by Abdelfattah Faouzi, Valérie Gaëlle Roullin
Published 2021-01-01