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Page 1
Progress in managing children with achondroplasia.
Taylor-Miller T, Savarirayan R. Taylor-Miller T, et al. Among authors: savarirayan r. Expert Rev Endocrinol Metab. 2024 Nov;19(6):479-486. doi: 10.1080/17446651.2024.2390416. Epub 2024 Aug 12. Expert Rev Endocrinol Metab. 2024. PMID: 39132812 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.
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.
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.
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.
Persistent growth-promoting effects of vosoritide in children with achondroplasia are accompanied by improvements in physical and social aspects of health-related quality of life.
Savarirayan R, Irving M, Wilcox WR, Bacino CA, Hoover-Fong JE, Harmatz P, Polgreen LE, Mohnike K, Prada CE, Kubota T, Arundel P, Leiva-Gea A, Rowell R, Low A, Sabir I, Huntsman-Labed A, Day J. Savarirayan R, et al. Genet Med. 2024 Dec;26(12):101274. doi: 10.1016/j.gim.2024.101274. Epub 2024 Sep 18. Genet Med. 2024. PMID: 39305160 Free article. Clinical Trial.
Sustained growth-promoting effects of vosoritide in children with achondroplasia from an ongoing phase 3 extension study.
Savarirayan R, Irving M, Wilcox WR, Bacino CA, Hoover-Fong JE, Harmatz P, Polgreen LE, Palm K, Prada CE, Kubota T, Arundel P, Kotani Y, Leiva-Gea A, Bober MB, Hecht JT, Legare JM, Lawrinson S, Low A, Sabir I, Huntsman-Labed A, Day JRS. Savarirayan R, et al. Med. 2024 Dec 30:100566. doi: 10.1016/j.medj.2024.11.019. Online ahead of print. Med. 2024. PMID: 39740666 Free article.
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.
223 results