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 POSITION STATEMENT Diagnostic and therapeutic recommendations for the treatment of hyperphenylalaninemia in patients 0–4 years of age Ania C. Muntau, Marcel du Moulin and Francois Feillet Orphanet Journal of Rare Diseases 2018, 13:173 | Published on: 29 September 2018 Full Text | PDF LETTER TO THE EDITOR Neutralizing anti-drug antibodies in Fabry disease have no obvious clinical impact? Malte Lenders, Boris Schmitz, Stefan-Martin Brand and Eva Brand Orphanet Journal of Rare Diseases 2018, 13:171 | Published on: 29 September 2018 Full Text | PDF RESEARCH Cost-effectiveness analysis of lumacaftor and ivacaftor combination for the treatment of patients with cystic fibrosis in the United States Dolly Sharma, Shan Xing, Yu-Ting Hung, Rachel N. Caskey, Maria L. Dowell and Daniel R. Touchette Orphanet Journal of Rare Diseases 2018, 13:172 | Published on: 29 September 2018 Full Text | PDF

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