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 REVIEW Epidemiological and clinical characteristics of symptomatic hereditary transthyretin amyloid polyneuropathy: a global case series Márcia Waddington-Cruz, Hartmut Schmidt, Marc F. Botteman, John A. Carter, Michelle Stewart, Markay Hopps, Shari Fallet and Leslie Amass Orphanet Journal of Rare Diseases 2019, 14:34 | Published on: 8 February 2019 Full Text | PDF RESEARCH Primary pulmonary lymphoma in children Xiaohui Wu, Chunju Zhou, Ling Jin, Hui Liu, Jinrong Liu and Shunying Zhao Orphanet Journal of Rare Diseases 2019, 14:35 | Published on: 8 February 2019 Full Text | PDF REVIEW Gastrointestinal Dysmotility in MNGIE: from thymidine phosphorylase enzyme deficiency to altered interstitial cells of Cajal Rana Yadak, Marjolein Breur and Marianna Bugiani Orphanet Journal of Rare Diseases 2019, 14:33 | Published on: 8 February 2019 Full Text | PDF POSITION STATEMENT Regulatory strategies for rare diseases under current global regulatory statutes: a discussion with stakeholders Andrew E. Mulberg, Christina Bucci-Rechtweg, Joseph Giuliano, David Jacoby, Franklin K. Johnson, Qing Liu, Deborah Marsden, Scott McGoohan, Robert Nelson, Nita Patel, Klaus Romero, Vikram Sinha, Sheela Sitaraman, John Spaltro and Vivian Kessler Orphanet Journal of Rare Diseases 2019, 14:36 | Published on: 8 February 2019 Full Text | PDF

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