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Fat infiltration in the thigh muscles is associated with symptomatic spinal stenosis and reduced physical functioning in adults with achondroplasia.
Fredwall SO, Linge J, de Vries O, Leinhard OD, Eggesbø HB, Weedon-Fekjær H, Petersson M, Widholm P, Månum G, Savarirayan R. Fredwall SO, et al. Among authors: savarirayan r. Orphanet J Rare Dis. 2023 Feb 22;18(1):35. doi: 10.1186/s13023-023-02641-5. Orphanet J Rare Dis. 2023. PMID: 36814258 Free PMC article.
New treatments for children with achondroplasia.
Savarirayan R, Hoover-Fong J, Yap P, Fredwall SO. Savarirayan R, et al. Lancet Child Adolesc Health. 2024 Apr;8(4):301-310. doi: 10.1016/S2352-4642(23)00310-3. Lancet Child Adolesc Health. 2024. PMID: 38485412 Review.
Australian guidelines for the management of children with achondroplasia.
Tofts LJ, Armstrong JA, Broley S, Carroll T, Ireland PJ, Koo M, Langdon K, McGregor L, McKenzie F, Mehta D, Savarirayan R, Tate T, Wesley A, Zankl A, Jenner M, Eyles M, Pacey V. Tofts LJ, et al. Among authors: savarirayan r. J Paediatr Child Health. 2023 Feb;59(2):229-241. doi: 10.1111/jpc.16290. Epub 2023 Jan 11. J Paediatr Child Health. 2023. PMID: 36628540 Free PMC article.
Vosoritide therapy in children with achondroplasia aged 3-59 months: a multinational, randomised, double-blind, placebo-controlled, phase 2 trial.
Savarirayan R, Wilcox WR, Harmatz P, Phillips J 3rd, Polgreen LE, Tofts L, Ozono K, Arundel P, Irving M, Bacino CA, Basel D, Bober MB, Charrow J, Mochizuki H, Kotani Y, Saal HM, Army C, Jeha G, Qi Y, Han L, Fisheleva E, Huntsman-Labed A, Day J. Savarirayan R, et al. Lancet Child Adolesc Health. 2024 Jan;8(1):40-50. doi: 10.1016/S2352-4642(23)00265-1. Epub 2023 Nov 18. Lancet Child Adolesc Health. 2024. PMID: 37984383 Clinical Trial.
Development of a Weight-Band Dosing Approach for Vosoritide in Children with Achondroplasia Using a Population Pharmacokinetic Model.
Qi Y, Chan ML, Mould DR, Larimore K, Fisheleva E, Cherukuri A, Day J, Savarirayan R, Irving M, Bacino CA, Hoover-Fong J, Ozono K, Mohnike K, Wilcox WR, Bober MB, Henshaw J. Qi Y, et al. Among authors: savarirayan r. Clin Pharmacokinet. 2024 May;63(5):707-719. doi: 10.1007/s40262-024-01371-6. Epub 2024 Apr 23. Clin Pharmacokinet. 2024. PMID: 38649657 Free PMC article.
Once-weekly TransCon CNP (navepegritide) in children with achondroplasia (ACcomplisH): a phase 2, multicentre, randomised, double-blind, placebo-controlled, dose-escalation trial.
Savarirayan R, Hoernschemeyer DG, Ljungberg M, Zarate YA, Bacino CA, Bober MB, Legare JM, Högler W, Quattrin T, Abuzzahab MJ, Hofman PL, White KK, Ma NS, Schnabel D, Sousa SB, Mao M, Smith A, Chakraborty M, Giwa A, Winding B, Volck B, Shu AD, McDonnell C. Savarirayan R, et al. EClinicalMedicine. 2023 Oct 2;65:102258. doi: 10.1016/j.eclinm.2023.102258. eCollection 2023 Nov. EClinicalMedicine. 2023. PMID: 37823031 Free PMC article.
Fibrodysplasia Ossificans Progressiva.
Akesson LS, Savarirayan R. Akesson LS, et al. Among authors: savarirayan r. 2020 Jun 11 [updated 2024 May 23]. In: Adam MP, Feldman J, Mirzaa GM, Pagon RA, Wallace SE, Bean LJH, Gripp KW, Amemiya A, editors. GeneReviews® [Internet]. Seattle (WA): University of Washington, Seattle; 1993–2024. 2020 Jun 11 [updated 2024 May 23]. In: Adam MP, Feldman J, Mirzaa GM, Pagon RA, Wallace SE, Bean LJH, Gripp KW, Amemiya A, editors. GeneReviews® [Internet]. Seattle (WA): University of Washington, Seattle; 1993–2024. PMID: 32525643 Free Books & Documents. Review.
Advances in the management of achondroplasia.
Savarirayan R. Savarirayan R. Nat Rev Endocrinol. 2024 May 13. doi: 10.1038/s41574-024-00994-w. Online ahead of print. Nat Rev Endocrinol. 2024. PMID: 38740894 No abstract available.
216 results