Orphanet Journal of Rare Diseases

The following new articles have just been published in Orphanet Journal of Rare Diseases

RESEARCH

A health state utility valuation study to assess the impact of treatment mode of administration in Gaucher disease

Monica Hadi, Paul Swinburn, Luba Nalysnyk, Alaa Hamed and Atul Mehta

Orphanet Journal of Rare Diseases 2018, 13:159 | Published on: 10 September 2018

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POSITION STATEMENT

Consensus statement on physical rehabilitation in children and adolescents with osteogenesis imperfecta

Brigitte Mueller, Raoul Engelbert, Frances Baratta-Ziska, Bart Bartels, Nicole Blanc, Evelise Brizola, Paolo Fraschini, Claire Hill, Caroline Marr, Lisa Mills, Kathleen Montpetit, Verity Pacey, Miguel Rodriguez Molina, Marleen Schuuring, Chantal Verhille, Olga de Vries…

Orphanet Journal of Rare Diseases 2018, 13:158 | Published on: 10 September 2018

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RESEARCH

TSC-associated neuropsychiatric disorders (TAND): findings from the TOSCA natural history study

Petrus J. de Vries, Elena Belousova, Mirjana P. Benedik, Tom Carter, Vincent Cottin, Paolo Curatolo, Maria Dahlin, Lisa D’Amato, Guillaume B. d’Augères, José C. Ferreira, Martha Feucht, Carla Fladrowski, Christoph Hertzberg, Sergiusz Jozwiak, J. Chris Kingswood, John A. Lawson…

Orphanet Journal of Rare Diseases 2018, 13:157 | Published on: 10 September 2018

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