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Page 1
Hereditary Transthyretin Amyloidosis in Patients Referred to a Genetic Testing Program.
Bhatt K, Delgado DH, Khella S, Bumma N, Karam C, Keller A, Rosen AM, Bozas A, Shea A, Towne MC, Polfus LM, Kaeser GE, Sanjurjo V, Shah KB. Bhatt K, et al. Among authors: khella s. J Am Heart Assoc. 2024 Dec 3;13(23):e033770. doi: 10.1161/JAHA.123.033770. Epub 2024 Nov 22. J Am Heart Assoc. 2024. PMID: 39575713 Free article.
Estimating Meaningful Differences in Measures of Neuropathic Impairment, Health-Related Quality of Life, and Nutritional Status in Patients With Hereditary Transthyretin Amyloidosis.
Folkvaljon F, Gertz M, Gillmore JD, Khella S, Masri A, Maurer MS, Cruz MW, Wixner J, Chen J, Reicher B, Kwoh J, Yarlas A, Berk JL. Folkvaljon F, et al. Among authors: khella s. Muscle Nerve. 2025 Jan;71(1):96-107. doi: 10.1002/mus.28299. Epub 2024 Nov 17. Muscle Nerve. 2025. PMID: 39552102 Free PMC article. Clinical Trial.
Author Correction: Precision targeting of autoantigen-specific B cells in muscle-specific tyrosine kinase myasthenia gravis with chimeric autoantibody receptor T cells.
Oh S, Mao X, Manfredo-Vieira S, Lee J, Patel D, Choi EJ, Alvarado A, Cottman-Thomas E, Maseda D, Tsao PY, Ellebrecht CT, Khella SL, Richman DP, O'Connor KC, Herzberg U, Binder GK, Milone MC, Basu S, Payne AS. Oh S, et al. Among authors: khella sl. Nat Biotechnol. 2024 Dec;42(12):1923. doi: 10.1038/s41587-024-02502-x. Nat Biotechnol. 2024. PMID: 39543316 Free PMC article. No abstract available.
Safety, tolerability, and efficacy of subcutaneous efgartigimod in patients with chronic inflammatory demyelinating polyradiculoneuropathy (ADHERE): a multicentre, randomised-withdrawal, double-blind, placebo-controlled, phase 2 trial.
Allen JA, Lin J, Basta I, Dysgaard T, Eggers C, Guptill JT, Gwathmey KG, Hewamadduma C, Hofman E, Hussain YM, Kuwabara S, Le Masson G, Leypoldt F, Chang T, Lipowska M, Lowe M, Lauria G, Querol L, Simu MA, Suresh N, Tse A, Ulrichts P, Van Hoorick B, Yamasaki R, Lewis RA, van Doorn PA; ADHERE Study Group. Allen JA, et al. Lancet Neurol. 2024 Oct;23(10):1013-1024. doi: 10.1016/S1474-4422(24)00309-0. Lancet Neurol. 2024. PMID: 39304241 Clinical Trial.
Switching from inotersen to eplontersen in patients with hereditary transthyretin-mediated amyloidosis with polyneuropathy: analysis from NEURO-TTRansform.
Conceição I, Berk JL, Weiler M, Kowacs PA, Dasgupta NR, Khella S, Chao CC, Attarian S, Kwoh TJ, Jung SW, Chen J, Viney NJ, Yu RZ, Gertz M, Masri A, Cruz MW, Coelho T. Conceição I, et al. Among authors: khella s. J Neurol. 2024 Oct;271(10):6655-6666. doi: 10.1007/s00415-024-12616-6. Epub 2024 Aug 13. J Neurol. 2024. PMID: 39138650 Free PMC article. Clinical Trial.
Eplontersen for Hereditary Transthyretin Amyloidosis With Polyneuropathy.
Coelho T, Marques W Jr, Dasgupta NR, Chao CC, Parman Y, França MC Jr, Guo YC, Wixner J, Ro LS, Calandra CR, Kowacs PA, Berk JL, Obici L, Barroso FA, Weiler M, Conceição I, Jung SW, Buchele G, Brambatti M, Chen J, Hughes SG, Schneider E, Viney NJ, Masri A, Gertz MR, Ando Y, Gillmore JD, Khella S, Dyck PJB, Waddington Cruz M; NEURO-TTRansform Investigators. Coelho T, et al. Among authors: khella s. JAMA. 2023 Oct 17;330(15):1448-1458. doi: 10.1001/jama.2023.18688. JAMA. 2023. PMID: 37768671 Free PMC article.
Impact of Vutrisiran on Quality of Life and Physical Function in Patients with Hereditary Transthyretin-Mediated Amyloidosis with Polyneuropathy.
Obici L, Ajroud-Driss S, Lin KP, Berk JL, Gillmore JD, Kale P, Koike H, Danese D, Aldinc E, Chen C, Vest J, Adams D; HELIOS-A Collaborators Study Group. Obici L, et al. Neurol Ther. 2023 Oct;12(5):1759-1775. doi: 10.1007/s40120-023-00522-4. Epub 2023 Jul 31. Neurol Ther. 2023. PMID: 37523143 Free PMC article.
59 results