The use or generation of biomedical data and existing medicines to discover and establish new treatments for patients with rare diseases – recommendations of the IRDiRC Data Mining and Repurposing Task Force

Autor: Noel T Southall, Madhusudan Natarajan, Lilian Pek Lian Lau, Anneliene Hechtelt Jonker, Benoît Deprez, Tim Guilliams, Lawrence Hunter, Carin MA Rademaker, Virginie Hivert, Diego Ardigò, on behalf of the IRDiRC Data Mining and Repurposing Task Force

Publikováno v: Orphanet Journal of Rare Diseases, Vol 14, Iss 1, Pp 1-8 (2019)

Abstract The number of available therapies for rare diseases remains low, as fewer than 6% of rare diseases have an approved treatment option. The International Rare Diseases Research Consortium (IRDiRC) set up the multi-stakeholder Data Mining and R

Externí odkaz: https://doaj.org/article/35fa88c515684ce8ae77763bca186370

Recommendations for the design of small population clinical trials

Autor: Anneliene H. Jonker, Nigel Stallard, Kit C.B. Roes, Ralf-Dieter Hilgers, Kristina Larsson, Lilian Pek Lian Lau, Ilan Irony, Simon Day

Publikováno v: Orphanet Journal of Rare Diseases, Vol 13, Iss 1, Pp 1-9 (2018)
Orphanet Journal of Rare Diseases
Orphanet Journal of Rare Diseases, 13(1). BioMed Central
Orphanet journal of rare diseases 13(1), 195 (2018). doi:10.1186/s13023-018-0931-2

Background: Orphan drug development faces numerous challenges, including low disease prevalence, patient population heterogeneity, and strong presence of paediatric patient populations. Consequently, clinical trials for orphan drugs are often smaller

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::33e215377865f2f4d2df1832025bb889
http://link.springer.com/article/10.1186/s13023-018-0931-2