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1,386 results
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Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy type 1 (STR1VE-EU): an open-label, single-arm, multicentre, phase 3 trial.
Mercuri E, Muntoni F, Baranello G, Masson R, Boespflug-Tanguy O, Bruno C, Corti S, Daron A, Deconinck N, Servais L, Straub V, Ouyang H, Chand D, Tauscher-Wisniewski S, Mendonca N, Lavrov A; STR1VE-EU study group. Mercuri E, et al. Lancet Neurol. 2021 Oct;20(10):832-841. doi: 10.1016/S1474-4422(21)00251-9. Lancet Neurol. 2021. PMID: 34536405
X-linked congenital adrenal hypoplasia: a case presentation.
Ouyang H, Chen B, Wu N, Li L, Du R, Qian M, Yu W, He Y, Liu X. Ouyang H, et al. BMC Endocr Disord. 2021 Jun 15;21(1):118. doi: 10.1186/s12902-021-00785-8. BMC Endocr Disord. 2021. PMID: 34130666 Free PMC article.
A long-term retaining molecular coating for corneal regeneration.
Zhang Y, Li C, Zhu Q, Liang R, Xie C, Zhang S, Hong Y, Ouyang H. Zhang Y, et al. Bioact Mater. 2021 May 5;6(12):4447-4454. doi: 10.1016/j.bioactmat.2021.04.032. eCollection 2021 Dec. Bioact Mater. 2021. PMID: 33997518 Free PMC article.
Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy in patients with two copies of SMN2 (STR1VE): an open-label, single-arm, multicentre, phase 3 trial.
Day JW, Finkel RS, Chiriboga CA, Connolly AM, Crawford TO, Darras BT, Iannaccone ST, Kuntz NL, Peña LDM, Shieh PB, Smith EC, Kwon JM, Zaidman CM, Schultz M, Feltner DE, Tauscher-Wisniewski S, Ouyang H, Chand DH, Sproule DM, Macek TA, Mendell JR. Day JW, et al. Lancet Neurol. 2021 Apr;20(4):284-293. doi: 10.1016/S1474-4422(21)00001-6. Epub 2021 Mar 17. Lancet Neurol. 2021. PMID: 33743238 Clinical Trial.
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