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Ai Huey Tan,Tien Lee Ong,Norlisah Ramli,Li Kuo Tan,Jia Lun Lim,Mohamad Addin Azhan,Azlina Ahmad Annuar,Khairul Azmi Ibrahim,Zariah Abdul-Aziz,Laurie J. Ozelius,Allison Brashear,Shen-Yang LIM 대한파킨슨병및이상운동질환학회 2019 Journal Of Movement Disorders Vol.12 No.2
Alternating hemiplegia of childhood (AHC) is a rare neurodevelopmentaldisorder with an incidence of one per million,characterized by paroxysmal alternating hemiplegia, eye movementabnormalities, dystonia, and epilepsy.1 In 2012, mutationsin the ATP1A3 gene encoding the Na+-K+-ATPase α3 subunit(originally discovered in rapid-onset dystonia-parkinsonism)were identified as the cause for AHC.1,2 However, the diagnosisof AHC is still often delayed or missed. We report a case of AHCdiagnosed in adulthood; to our knowledge, this is the first reportin a person of Malay ethnicity.
Jinlin Hou,Edward Gane,Rozalina Balabanska,Wenhong Zhang,Jiming Zhang,Tien Huey Lim,Qing Xie,Chau-Ting Yeh,Sheng-Shun Yang,Xieer Liang,Piyawat Komolmit,Apinya Leerapun,Zenghui Xue,Ethan Chen,Yuchen Zh 대한간학회 2024 Clinical and Molecular Hepatology(대한간학회지) Vol.30 No.2
Background/Aims: Four-week treatment of linvencorvir (RO7049389) was generally safe and well tolerated, and showed anti-viral activity in chronic hepatitis B (CHB) patients. This study evaluated the efficacy, safety, and pharmacokinetics of 48-week treatment with linvencorvir plus standard of care (SoC) in CHB patients. Methods: This was a multicentre, non-randomized, non-controlled, open-label phase 2 study enrolling three cohorts: nucleos(t)ide analogue (NUC)-suppressed patients received linvencorvir plus NUC (Cohort A, n=32); treatment-naïve patients received linvencorvir plus NUC without (Cohort B, n=10) or with (Cohort C, n=30) pegylated interferon-α (Peg-IFN-α). Treatment duration was 48 weeks, followed by NUC alone for 24 weeks. Results: 68 patients completed the study. No patient achieved functional cure (sustained HBsAg loss and unquantifiable HBV DNA). By Week 48, 89% of treatment-naïve patients (10/10 Cohort B; 24/28 Cohort C) reached unquantifiable HBV DNA. Unquantifiable HBV RNA was achieved in 92% of patients with quantifiable baseline HBV RNA (14/15 Cohort A, 8/8 Cohort B, 22/25 Cohort C) at Week 48 along with partially sustained HBV RNA responses in treatment-naïve patients during follow-up period. Pronounced reductions in HBeAg and HBcrAg were observed in treatment-naïve patients, while HBsAg decline was only observed in Cohort C. Most adverse events were grade 1–2, and no linvencorvir-related serious adverse events were reported. Conclusions: 48-week linvencorvir plus SoC was generally safe and well tolerated, and resulted in potent HBV DNA and RNA suppression. However, 48-week linvencorvir plus NUC with or without Peg-IFN did not result in the achievement of functional cure in any patient.