Orphanet Journal of Rare Diseases | Home page

Orphanet Journal of Rare Diseases | Home page





Orphanet Journal of Rare Diseases

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1. 
   
2. REVIEW

   Minimal clinically important difference for the 6-min walk test: literature review and application to Morquio A syndrome

   Rudolf Schrover, Kathryn Evans, Roberto Giugliani, Ian Noble and Kaustuv Bhattacharya

   Published on: 26 April 2017
3. REVIEW

   The burden, epidemiology, costs and treatment for Duchenne muscular dystrophy: an evidence review

   S. Ryder, R. M. Leadley, N. Armstrong, M. Westwood, S. de Kock, T. Butt, M. Jain and J. Kleijnen

   Published on: 26 April 2017
4. RESEARCH

   Extrastriatal changes in patients with late-onset glutaric aciduria type I highlight the risk of long-term neurotoxicity

   Nikolas Boy, Jana Heringer, Renate Brackmann, Olaf Bodamer, Angelika Seitz, Stefan Kölker and Inga Harting

   Published on: 24 April 2017
5. RESEARCH

   Determinants of orphan drugs prices in France: a regression analysis

   Daria Korchagina, Aurelie Millier, Anne-Lise Vataire, Samuel Aballea, Bruno Falissard and Mondher Toumi

   Published on: 21 April 2017
6. RESEARCH

   Capture-recapture methodology to study rare conditions using surveillance data for fragile X syndrome and muscular dystrophy

   Michael G. Smith, Julie Royer, Joshua Mann, Suzanne McDermott and Rodolfo Valdez

   Published on: 21 April 2017

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