Orphanet Journal of Rare Diseases | Home page

Orphanet Journal of Rare Diseases | Home page

Orphanet Journal of Rare Diseases

The following new articles have just been published in Orphanet Journal of Rare Diseases RESEARCH An online survey on burden of illness among families with post-stem cell transplant mucopolysaccharidosis type I children in the United States Therese Conner, Francesca Cook, Vivian Fernandez, Karen Rascati and Vanessa Rangel-Miller Orphanet Journal of Rare Diseases 2019, 14:48 | Published on: 18 February 2019 Full Text | PDF RESEARCH Patient access to orphan drugs in France Marion Bourdoncle, Blandine Juillard-Condat and Florence Taboulet Orphanet Journal of Rare Diseases 2019, 14:47 | Published on: 18 February 2019 Full Text | PDF RESEARCH Early progression of Krabbe disease in patients with symptom onset between 0 and 5 months Maria L. Beltran-Quintero, Nicholas A. Bascou, Michele D. Poe, David A. Wenger, Carlos A. Saavedra-Matiz, Matthew J. Nichols and Maria L. Escolar Orphanet Journal of Rare Diseases 2019, 14:46 | Published on: 18 February 2019 Full Text | PDF RESEARCH The cumulative incidence and trends of rare diseases in South Korea: a nationwide study of the administrative data from the National Health Insurance Service database from 2011–2015 Sung-Shil Lim, Wanhyung Lee, Yeong-Kwang Kim, Jihyun Kim, Jong Heon Park, Bo Ram Park and Jin-Ha Yoon Orphanet Journal of Rare Diseases 2019, 14:49 | Published on: 18 February 2019 Full Text | PDF RESEARCH Requirement for etoposide in the treatment of pregnancy related hemophagocytic lymphohistiocytosis: a multicenter retrospective study Yue Song, Zhao Wang, Zengping Hao, Lihong Li, Junli Lu, Hongjun Kang, Yanping Lu, Yanqin You, Lijuan Li, Qingyun Chen and Bo Chen Orphanet Journal of Rare Diseases 2019, 14:50 | Published on: 18 February 2019 Full Text | PDF

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