News
Authors: J-H Tsai, N L Crossnohere, T Strong, J F P Bridges Published: MDM policy & practice, 2021 View publication Background Prader-Willi syndrome (PWS) is a rare neurodevelopmental disorder causing quality of...
Authors: M Cheung, A J Rylands, A Williams, K Bailey, J Bubbear Published: Journal of the Endocrine Society, 2021 View publication Context X-linked hypophosphatemia (XLH) is a rare, genetic phosphate-wasting disease resulting...
The 11th European Congress on Rare Diseases (ECRD) was held virtually over the week following the Glastonbury festival. Like the fabled festival of contemporary performing arts, there are often moments that define...
This article was first published in July 2022 in partnership with the Medical Affairs Professional Society (MAPS), available here. As complex treatments are developed for ever-more-targeted patient populations and second- and third-generation...
Authors: V L Gowda, M Fernandez, M Prasad, A-M Childs, I Hughes, S Tirupathi, C G E L De Goede, D O'Rourke, D Parasuraman, T Willis, S Saberian, I Davidson Published: Archives...
The Digital Health World Congress 2022 took place in London from 17-18, May. The congress was attended by Terryn Archer and Frankie Evans from OPEN Health’s Technology Solutions team, who gathered insights...
Authors: S Minisola, A Barlassina, S-A Vincent, S Wood, A Williams Published: Osteoporosis International, 2022 View publication Summary This study reviews publications to describe the signs, symptoms and impact of tumour-induced osteomalacia...
Authors: J Radford, P McKay, R Malladi, R Johnson, A Bloor, F Percival, A Sureda, K S Peggs Published: Bone Marrow Transplantation, 2017 View publication Although many patients with Hodgkin lymphoma (HL)...
Authors: M J Postma, D Noone, M H Rozenbaum, J A Carter, M F Botteman, E Fenwick, L P Garrison Published: Orphanet Journal of Rare Diseases, 2022 View publication Abstract Conventional cost-effectiveness...