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The IRDiRC Chrysalis Task Force: making rare disease research attractive to companies.
Beaverson KL, Julkowska D, Letinturier MCV, Aartsma-Rus A, Austin J, Bueren J, Frost S, Hamamura M, Larkindale J, LaRosa G, Magenheim R, Merico A, Pasmooij AMG, Pirard V, Ekow Thomford N, Wada M, Wong-Rieger D, Hartman AL. Beaverson KL, et al. Among authors: wong rieger d. Ther Adv Rare Dis. 2023 Jul 29;4:26330040231188979. doi: 10.1177/26330040231188979. eCollection 2023 Jan-Dec. Ther Adv Rare Dis. 2023. PMID: 37529076 Free PMC article.
Barriers and facilitators to designing, maintaining, and utilizing rare disease patient registries: a scoping review protocol.
Stratton C, Taylor A, Konstantinidis M, McNiven V, Kannu P, Gill P, Stedman I, Veroniki AA, Offringa M, Potter B, Wong-Rieger D, Adams J, Hodgkinson K, Elliott AM, Neville A, Faughnan M, Dyack S, Zhelnov P, Daly-Cyr J, McGowan J, Straus S, Smith M, Rosella L, Tricco AC. Stratton C, et al. Among authors: wong rieger d. JBI Evid Synth. 2024 Oct 21. doi: 10.11124/JBIES-24-00091. Online ahead of print. JBI Evid Synth. 2024. PMID: 39428992
Strengthening health systems for access to gene therapy in rare genetic disorders.
Bhatia S, Le Cam Y, Carrion J, Diamond L, Fennessy P, Gassman S, Gutzwiller F, Kagan S, Pankevich D, Young Maloney J, Mahadev N, Schulz M, Wong-Rieger D, Morgese P. Bhatia S, et al. Among authors: wong rieger d. Mol Ther Methods Clin Dev. 2024 Mar 13;32(2):101220. doi: 10.1016/j.omtm.2024.101220. eCollection 2024 Jun 13. Mol Ther Methods Clin Dev. 2024. PMID: 38516694 Free PMC article. No abstract available.
Developing a Framework of Cost Elements of Socioeconomic Burden of Rare Disease: A Scoping Review.
Currie GR, Gerber B, Lorenzetti D, MacDonald K, Benseler SM, Bernier FP, Boycott KM, Carias KV, Hamelin B, Hayeems RZ, LeBlanc C, Twilt M, van Rooijen G, Wong-Rieger D, Yeung RSM, Marshall DA. Currie GR, et al. Among authors: wong rieger d. Pharmacoeconomics. 2023 Jul;41(7):803-818. doi: 10.1007/s40273-023-01262-x. Epub 2023 Apr 7. Pharmacoeconomics. 2023. PMID: 37029233 Review.
Research on rare diseases: ten years of progress and challenges at IRDiRC.
Monaco L, Zanello G, Baynam G, Jonker AH, Julkowska D, Hartman AL, O'Connor D, Wang CM, Wong-Rieger D, Pearce DA. Monaco L, et al. Among authors: wong rieger d. Nat Rev Drug Discov. 2022 May;21(5):319-320. doi: 10.1038/d41573-022-00019-z. Nat Rev Drug Discov. 2022. PMID: 35079160 Free PMC article. No abstract available.
Potential approaches for the pricing of cancer medicines across Europe to enhance the sustainability of healthcare systems and the implications.
Godman B, Hill A, Simoens S, Selke G, Selke Krulichová I, Zampirolli Dias C, Martin AP, Oortwijn W, Timoney A, Gustafsson LL, Voncina L, Kwon HY, Gulbinovic J, Gotham D, Wale J, Cristina Da Silva W, Bochenek T, Allocati E, Kurdi A, Ogunleye OO, Meyer JC, Hoxha I, Malaj A, Hierländer C, Sauermann R, Hamelinck W, Petrova G, Laius O, Langner I, Yfantopoulos J, Joppi R, Jakupi A, Greiciute-Kuprijanov I, Vella Bonanno P, Piepenbrink JH, de Valk V, Wladysiuk M, Marković-Peković V, Mardare I, Fürst J, Tomek D, Obach Cortadellas M, Zara C, Pontes C, McTaggart S, Laba TL, Melien Ø, Wong-Rieger D, Bae S, Hill R. Godman B, et al. Among authors: wong rieger d. Expert Rev Pharmacoecon Outcomes Res. 2021 Aug;21(4):527-540. doi: 10.1080/14737167.2021.1884546. Epub 2021 Mar 11. Expert Rev Pharmacoecon Outcomes Res. 2021. PMID: 33535841 Free article.
46 results