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Orphanet Journal of Rare Diseases|December 13, 2019
An analysis of orphan medicine expenditure in Europe: is it sustainable?Jorge Mestre-Ferrandiz, Christina Palaska, Tom Kelly, et al.
Orphanet Journal of Rare Diseases|March 7, 2020
Cardiovascular progenitor cells and tissue plasticity are reduced in a myocardium affected by Becker muscular dystrophyMartin Pesl, Sarka Jelinkova, Guido Caluori, et al.
Orphanet Journal of Rare Diseases|February 29, 2020
No friends 1Anna-Lucia Koerling
Orphanet Journal of Rare Diseases|March 14, 2020
Health, wellbeing and lived experiences of adults with SMA: a scoping systematic reviewHamish W Y Wan, Kate A Carey, Arlene D'Silva, et al.
Orphanet Journal of Rare Diseases|December 12, 2018
Determining the value contribution of selexipag for the treatment of pulmonary arterial hypertension (PAH) in Spain using reflective multi-criteria decision analysis (MCDA)Alberto Jiménez, Arantza Ais, Amélie Beaudet, et al.
Orphanet Journal of Rare Diseases|January 12, 2019
Estimating the clinical cost of drug development for orphan versus non-orphan drugsKavisha Jayasundara, Aidan Hollis, Murray Krahn, et al.
Orphanet Journal of Rare Diseases|January 12, 2019
DeepNEU: cellular reprogramming comes of age - a machine learning platform with application to rare diseases researchWayne R Danter
Orphanet Journal of Rare Diseases|December 5, 2019
Developmental hypomyelination in Wolfram syndrome: new insights from neuroimaging and gene expression analysesAmjad Samara, Rachel Rahn, Olga Neyman, et al.
Orphanet Journal of Rare Diseases|November 30, 2019
Poor prognostic factors in patients with newly diagnosed intestinal Adamantiades-Behçet's disease in the Shanghai Adamantiades-Behçet's disease database: a prospective cohort studyLiang Zhang, Yun Tian, Jing-Fen Ye, et al.
Orphanet Journal of Rare Diseases|November 30, 2019
Adult cognitive outcomes in phenylketonuria: explaining causes of variability beyond average Phe levelsCristina Romani, Filippo Manti, Francesca Nardecchia, et al.
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