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'Advocacy groups are the connectors': Experiences and contributions of rare disease patient organization leaders in advanced neurotherapeutics.
Nguyen CQ, Kariyawasam D, Alba-Concepcion K, Grattan S, Hetherington K, Wakefield CE, Woolfenden S, Dale RC, Palmer EE, Farrar MA. Nguyen CQ, et al. Among authors: kariyawasam d. Health Expect. 2022 Dec;25(6):3175-3191. doi: 10.1111/hex.13625. Epub 2022 Oct 28. Health Expect. 2022. PMID: 36307981 Free PMC article.
New and developing therapies in spinal muscular atrophy.
Kariyawasam D, Carey KA, Jones KJ, Farrar MA. Kariyawasam D, et al. Paediatr Respir Rev. 2018 Sep;28:3-10. doi: 10.1016/j.prrv.2018.03.003. Epub 2018 Apr 5. Paediatr Respir Rev. 2018. PMID: 29703692 Review.
Onasemnogene abeparvovec in spinal muscular atrophy: an Australian experience of safety and efficacy.
D'Silva AM, Holland S, Kariyawasam D, Herbert K, Barclay P, Cairns A, MacLennan SC, Ryan MM, Sampaio H, Smith N, Woodcock IR, Yiu EM, Alexander IE, Farrar MA. D'Silva AM, et al. Among authors: kariyawasam d. Ann Clin Transl Neurol. 2022 Mar;9(3):339-350. doi: 10.1002/acn3.51519. Epub 2022 Feb 16. Ann Clin Transl Neurol. 2022. PMID: 35170254 Free PMC article.
97 results