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Burden and Treatment of Achondroplasia: A Systematic Literature Review.
Murton MC, Drane ELA, Goff-Leggett DM, Shediac R, O'Hara J, Irving M, Butt TJ. Murton MC, et al. Among authors: butt tj. Adv Ther. 2023 Sep;40(9):3639-3680. doi: 10.1007/s12325-023-02549-3. Epub 2023 Jun 29. Adv Ther. 2023. PMID: 37382866 Free PMC article. Review.
Literature review and expert opinion on the impact of achondroplasia on medical complications and health-related quality of life and expectations for long-term impact of vosoritide: a modified Delphi study.
Savarirayan R, Baratela W, Butt T, Cormier-Daire V, Irving M, Miller BS, Mohnike K, Ozono K, Rosenfeld R, Selicorni A, Thompson D, White KK, Wright M, Fredwall SO. Savarirayan R, et al. Orphanet J Rare Dis. 2022 Jun 13;17(1):224. doi: 10.1186/s13023-022-02372-z. Orphanet J Rare Dis. 2022. PMID: 35698202 Free PMC article.
Lifetime impact of achondroplasia study in Europe (LIAISE): findings from a multinational observational study.
Maghnie M, Semler O, Guillen-Navarro E, Selicorni A, Heath KE, Haeusler G, Hagenäs L, Merker A, Leiva-Gea A, González VL, Raimann A, Rehberg M, Santos-Simarro F, Ertl DA, Gregersen PA, Onesimo R, Landfeldt E, Jarrett J, Quinn J, Rowell R, Pimenta J, Cohen S, Butt T, Shediac R, Mukherjee S, Mohnike K. Maghnie M, et al. Orphanet J Rare Dis. 2023 Mar 15;18(1):56. doi: 10.1186/s13023-023-02652-2. Orphanet J Rare Dis. 2023. PMID: 36922864 Free PMC article.
The economics of vision impairment and its leading causes: A systematic review.
Marques AP, Ramke J, Cairns J, Butt T, Zhang JH, Jones I, Jovic M, Nandakumar A, Faal H, Taylor H, Bastawrous A, Braithwaite T, Resnikoff S, Khaw PT, Bourne R, Gordon I, Frick K, Burton MJ. Marques AP, et al. EClinicalMedicine. 2022 Mar 22;46:101354. doi: 10.1016/j.eclinm.2022.101354. eCollection 2022 Apr. EClinicalMedicine. 2022. PMID: 35340626 Free PMC article. Review.
Investigating health-related quality of life in rare diseases: a case study in utility value determination for patients with CLN2 disease (neuronal ceroid lipofuscinosis type 2).
Gissen P, Specchio N, Olaye A, Jain M, Butt T, Ghosh W, Ruban-Fell B, Griffiths A, Camp C, Sisic Z, Schwering C, Wibbeler E, Trivisano M, Lee L, Nickel M, Mortensen A, Schulz A. Gissen P, et al. Orphanet J Rare Dis. 2021 May 12;16(1):217. doi: 10.1186/s13023-021-01829-x. Orphanet J Rare Dis. 2021. PMID: 33980287 Free PMC article.
26 results