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Drug for treating CKLD5 deficiency related epileptic seizures: ganaxolone
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Yuan-yuan LI1, 2, Qun GU1, Lin HUANG1
Chinese Journal of New Drugs and Clinical Remedies | 2024, 43(6) : 432 - 436
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Chinese Journal of New Drugs and Clinical Remedies | 2024, 43(6): 432-436
New Drug Introduction
Drug for treating CKLD5 deficiency related epileptic seizures: ganaxolone
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Yuan-yuan LI1, 2, Qun GU1, Lin HUANG1
Affiliations
  • 1.Department of Pharmacy, People’s Hospital of Peking University, BEIJING 100044, China
  • 2.School of Basic Medicine and Clinical Pharmacy, China Pharmaceutical University, Nanjing JIANGSU 211198, China
Published: 2024-06-25 doi: 10.14109/j.cnki.xyylc.2024.06.07
Outline
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Ganaxolone is an oral GABAA receptor positive variant modulator developed by Marinas Pharmaceuticals in the United States, and it can specifically modulate GABAA receptors in the central nervous system to enhance temporal and tonic inhibition for seizure control. In March 2022, the U.S. Food and Drug Administration approved it as the first drug to treat cyclin-dependent kinase-like 5 deficiency based on the results of a major phase Ⅲ clinical trial. Clinical trials have shown that ganaxolone is well tolerated, and the treatment regimen based on this drug is effective in controlling seizure frequency of patients and improving their quality of life.

ganaxolone  /  cyclin-dependent kinase-like 5  /  epilepsy  /  receptors, GABA-A  /  treatment outcome  /  safety
Yuan-yuan LI, Qun GU, Lin HUANG. Drug for treating CKLD5 deficiency related epileptic seizures: ganaxolone[J]. Chinese Journal of New Drugs and Clinical Remedies, 2024 , 43 (6) : 432 -436 . DOI: 10.14109/j.cnki.xyylc.2024.06.07
Year 2024 volume 43 Issue 6
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Article Info
doi: 10.14109/j.cnki.xyylc.2024.06.07
  • Receive Date:2023-01-17
  • Online Date:2026-03-20
  • Published:2024-06-25
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History
  • Received:2023-01-17
  • Accepted:2024-02-02
Affiliations
    1.Department of Pharmacy, People’s Hospital of Peking University, BEIJING 100044, China
    2.School of Basic Medicine and Clinical Pharmacy, China Pharmaceutical University, Nanjing JIANGSU 211198, China
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表12种不同金属材料的力学参数

Family
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Number of
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种数
Number of
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鹅膏菌科Amanitaceae 2 11 5.26 鹅膏菌属 Amanita 10 4.78
小菇科 Mycenaceae 2 12 5.74 丝盖伞属 Inocybe 5 2.39
多孔菌科 Polyporaceae 8 14 6.70 蜡蘑属 Laccaria 5 2.39
红菇科 Russulaceae 3 23 11.00 小皮伞属 Marasmius 6 2.87
小菇属 Mycena 11 5.26
光柄菇属 Pluteus 5 2.39
红菇属 Russula 17 8.13
栓菌属 Trametes 5 2.39
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