Orphanet Journal of Rare Diseases | Home page

Orphanet Journal of Rare Diseases | Home page





Orphanet Journal of Rare Diseases

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3. RESEARCH

   Transcranial pulsed ultrasound facilitates brain uptake of laronidase in enzyme replacement therapy for Mucopolysaccharidosis type I disease

   Yu-Hone Hsu, Ren-Shyan Liu, Win-Li Lin, Yeong-Seng Yuh, Shuan-Pei Lin and Tai-Tong Wong

   Published on: 8 June 2017
4. RESEARCH

   Treatment with long-acting lanreotide autogel in early infancy in patients with severe neonatal hyperinsulinism

   Heike Corda, Sebastian Kummer, Alena Welters, Norbert Teig, Dirk Klee, Ertan Mayatepek and Thomas Meissner

   Published on: 2 June 2017
5. RESEARCH

   The cost of severe haemophilia in Europe: the CHESS study

   Jamie O’Hara, David Hughes, Charlotte Camp, Tom Burke, Liz Carroll and Daniel-Anibal Garcia Diego

   Published on: 31 May 2017
6. LETTER TO THE EDITOR

   The work experience of a patient affected by Williams Syndrome: a pilot project at the Bambino Gesù Children’s Hospital

   Francesca De Lorenzo, Marina Macchiaiolo, Carla Maria Carlevaris and Andrea Bartuli

   Published on: 31 May 2017
7. RESEARCH

   Tocilizumab in the treatment of twelve cases with aa amyloidosis secondary to familial mediterranean fever

   Serdal Ugurlu, Aysa Hacioglu, Yasaman Adibnia, Vedat Hamuryudan and Huri Ozdogan

   Published on: 30 May 2017

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