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Orphanet Journal of Rare Diseases|December 4, 2019
Endocrine and metabolic disorders in patients with Gaucher disease type 1: a reviewMałgorzata Kałużna, Isabella Trzeciak, Katarzyna Ziemnicka, et al.
Orphanet Journal of Rare Diseases|February 14, 2020
Machine learning application for development of a data-driven predictive model able to investigate quality of life scores in a rare diseaseOttavia Spiga, Vittoria Cicaloni, Cosimo Fiorini, et al.
Orphanet Journal of Rare Diseases|February 15, 2020
Adverse event rates and economic burden associated with purine nucleoside analogs in patients with hairy cell leukemia: a US population-retrospective claims analysisNarendranath Epperla, Melissa Pavilack, Temitope Olufade, et al.
Orphanet Journal of Rare Diseases|January 23, 2020
A systematic review on the burden of illness in individuals with tuberous sclerosis complex (TSC)Johann Philipp Zöllner, David Neal Franz, Christoph Hertzberg, et al.
Orphanet Journal of Rare Diseases|January 10, 2020
Genetic profiling and cardiovascular phenotypic spectrum in a Chinese cohort of Loeys-Dietz syndrome patientsHang Yang, Yanyun Ma, Mingyao Luo, et al.
Orphanet Journal of Rare Diseases|January 12, 2020
Management and current status of spinal muscular atrophy: a retrospective multicentre claims database analysisJosep Darbà
Orphanet Journal of Rare Diseases|February 6, 2019
Safety of thalidomide and bevacizumab in patients with hereditary hemorrhagic telangiectasiaElisabetta Buscarini, Luisa Maria Botella, Urban Geisthoff, et al.
Orphanet Journal of Rare Diseases|January 23, 2019
Tracking sex-dependent differences in a mouse model of CLN6-Batten diseaseMcKayla J Poppens, Jacob T Cain, Tyler B Johnson, et al.
Orphanet Journal of Rare Diseases|February 10, 2019
Gastrointestinal Dysmotility in MNGIE: from thymidine phosphorylase enzyme deficiency to altered interstitial cells of CajalRana Yadak, Marjolein Breur, Marianna Bugiani
Orphanet Journal of Rare Diseases|February 10, 2019
Regulatory strategies for rare diseases under current global regulatory statutes: a discussion with stakeholdersAndrew E Mulberg, Christina Bucci-Rechtweg, Joseph Giuliano, et al.
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