1.Advances in Disease-Modifying Therapy of Neuromyelitis Optic Spectrum Disorders
Xuanling ZHANG ; Chaoyang CHEN ; Ran WEI ; Ying ZHOU
JOURNAL OF RARE DISEASES 2024;3(2):246-251
Neuromyelitis optic spectrum disorders(NMOSD)is a serious inflammatory demyelinating disease.Clinical treatment of NMOSD mainly involves acute phase treatment and remission phase prevention treatment.Disease-modifying therapy(DMT)in remission phase of NMOSD can effectively reduce disease re-currence and delay disability progression.At present,the drugs commonly used for DMT at home and abroad in-clude classical immunosuppression,B-cell surface antigen monoclonal antibody,complement targeting mono-clonal antibody,IL-6 receptor-targeting monoclonal antibody,etc.This paper mainly reviews the DMT drugs ef-ficacy,safety,as well as the marketing status of NMOSD.
2.Advances in the Adjunctive Treatment of Lennox-Gastaut Syndrome with Clobazam and Cannabidiol
Jingtian SHI ; Chaoyang CHEN ; Ting YANG ; Ran WEI ; Xuanling ZHANG ; Zining WANG ; Xiaojuan HU ; Ying ZHOU
JOURNAL OF RARE DISEASES 2024;3(2):252-259
Lennox-Gastaut syndrome(LGS)is a severe,epileptic encephalopathy.In recent years,a variety of drugs have been approved for the treatment of LGS.The U.S.Food and Drug Administration ap-proved clobazam and cannabidiol as adjunctive therapy for LGS in October 2011 and June 2018,respectively.This article provides an overview of clobazam and cannabidiol,including their chemical structures,pharmaco-logical actions,curative effects,safety profile,drug interactions,to introduce the current state of research and the achievements of both drugs.
3.Progress in the Diagnosis and Treatment of Infantile Epileptic Spasm Syndrome
Lu ZHENG ; Ting YANG ; Chaoyang CHEN ; Ran WEI ; Xuanling ZHANG ; Yijie MA ; Ying ZHOU
JOURNAL OF RARE DISEASES 2024;3(2):260-268
Infantile epileptic spasm syndrome(IESS)is a new concept proposed recently.IESS is a unique and age-specific refractory epilepsy syndrome.The recent advances in molecular biology,neuroimmunol-ogy and the in-depth study of anti-epileptic mechanism in antiepileptic drugs have led to the achievements in the definition and treatment of infantile epileptic spasm.At present,the use of traditional antiepileptic drugs is de-creasing,while the use of new antiepileptic drugs is increasing.In this paper,based on the relevant literature in recent years,the authors discuss the pathogenesis,epidemiology,etiology,diagnosis,treatment,therapeutic drugs,clinical progress,efficacy,and safety of infantile epileptic spasm,hoping to introduce the latest status in research and achievements of IESS.
4.Pozelimab,a new generation of C5 complement inhibitors for CHAPLE disease
Xuan-Ling ZHANG ; Chao-Yang CHEN ; Ran WEI ; Ying ZHOU
The Chinese Journal of Clinical Pharmacology 2024;40(2):276-278
On August 18,2023,the U.S.Food and Drug Administration(FDA)officially approved Regeneron's Pozelimab-bbfg(Veopoz?)for the treatment of complement factor H-related protein 5 deficiency type protein-losing enteropathy(CHAPLE)disease in children aged 1 and above,as well as adult patients.CHAPLE disease is a rare condition.Pazelimab monoclonal antibody is a recombinant monoclonal antibody(immunoglobulin G4 isotype).This antibody works by binding to the terminal complement protein C5,thereby preventing the cleavage of C5 into C5a(an anaphylatoxin)and C5b,and inhibit the activation of the terminal complement pathway.This article provides an overview of Pozelimab-bbfg,including its pharmacological research,pharmacokinetics,clinical studies,safety profile,and more,to introduce the current state of research and the achievements made with this drug.
5.Palovarotene:A new drug for fibrodysplasia ossificans progressive
Ran WEI ; Chao-Yang CHEN ; Xuan-Ling ZHANG ; Ying ZHOU
The Chinese Journal of Clinical Pharmacology 2024;40(5):745-748
Progressive ossifying fibrous dysplasia(FOP)is a rare,hereditary,and progressive connective tissue disease characterized by heterotopic ossification(HO)formation in muscles,joints,tendons,and ligaments,leading to major joint stiffness,limited movement,deformity,and severe disability in patients.In August 2023,Palovarotene was approved by the US Food and Drug Administration(FDA)to reduce the formation of HO in adults and children aged 8 years and above for females and 10 years and above for males with FOP.It is currently the only approved curable drug worldwide.Palovarotene is a selective retinoic acid receptor γ(RAR-γ)agonist that reduces the formation of HO by inhibiting bone morphogenetic protein and SMAD 1/5/8 signaling.The pharmacological study,pharmacokinetics,clinical research,and safety are reviewed to comprehensively introduce Palovarotene.
6.68 Ga-DOTATOC,a new diagnostic agent for localization of somatostatin receptor positive neuroendocrine tumors
Si-Miao ZHAO ; Chao-Yang CHEN ; Ran WEI ; Xuan-Ling ZHANG ; Ying ZHOU
The Chinese Journal of Clinical Pharmacology 2024;40(11):1654-1657
68Ga-DOTATOC injection is a radiopharmaceutical agent for positron emission tomography localization of somatostatin receptor positive neuroendocrine tumors(NETs)in adult and pediatric patients.68 Ga-DOTATOC binds to cells that express somatostatin receptors(SSTRs),including malignant neuroendocrine cells that overexpress SSTR2 receptor.Gallium-68 is a radionuclide used in positron emission tomography for tumor diagnosis.This paper introduces its the mechanism of action,pharmacokinetics,usage and dosage,clinical evaluation,safety and use in specific populations.
7.Pafolacianine,optical imaging agents for intraoperative tumor detection
Zhao WANG ; Chao-Yang CHEN ; Ran WEI ; Xuan-Ling ZHANG ; Xiao-Juan HU ; Ying ZHOU
The Chinese Journal of Clinical Pharmacology 2024;40(11):1658-1660
Pafolacianine is a fluorescent agent that specifically targets folate receptors,employed in the treatment of adult ovarian cancer patients to support the detection of malignant growths during surgical interventions.Pafolacianine binds to cancer cells expressing folate receptors,accumulating within folate-receptor-positive tumor tissue through receptor-mediated endocytosis.It can be excited by near-infrared fluorescence imaging,facilitating the surgical removal of tumors during procedures.This article gives an introduction to the mechanism of action,pharmacokinetics,clinical studies,and safety aspects of Pafolacianine.
8.Gadopiclenol,a new radiological diagnostic drug used in magnetic resonance imaging
Lu ZHENG ; Ting YANG ; Chao-Yang CHEN ; Ran WEI ; Xuan-Ling ZHANG ; Jing-Zhong DENG ; Ying ZHOU
The Chinese Journal of Clinical Pharmacology 2024;40(11):1661-1664
Gadopiclenol was used in adults and pediatric patients 2 years of age and older during magnetic resonance imaging(MRI)to detect and view lesions of the central nervous system(brain,spine,and associated tissues)and body(head and neck,chest,abdomen,pelvis,and musculoskeletal system)with abnormal vascular properties.Gadopiclenol is a new type of macrocyclic gadolinium-based contrast agent(GBCA).In this article,the molecular structure,principle of action,pharmacodynamics,pharmacokinetics,clinical studies,safety and other aspects of Gadopiclenol were reviewed,in order to introduce the current research status and existing achievements of Gadopiclenol.
9.Copper Cu 64 dotatate,a new diagnostic agent for somatostatin receptor-positive neuroendocrine tumors
Ming-Zhu XU ; Chao-Yang CHEN ; Ran WEI ; Xuan-Ling ZHANG ; Ying ZHOU
The Chinese Journal of Clinical Pharmacology 2024;40(12):1832-1834
On September 3,2020,the U.S.Food and Drug Administration(FDA)granted formal approval to Copper Cu 64 dotatate(commercially known as Detectnet),for its pivotal role in diagnosing somatostatin receptor(SSTR)positive neuroendocrine tumors(NETs)in adult patients.This innovative diagnostic agent,Copper Cu 64 dotatate,is a sophisticated radionuclide that selectively targets somatostatin receptors,facilitating the emission of positron(β+)radionuclides in a quantity conducive for high-resolution positron emission tomography(PET)imaging.This review delves into the intricate mechanisms of action,the dynamic pharmacokinetics,comprehensive clinical studies,and the safety profile of Copper Cu 64 dotatate,highlighting its significance in the realm of medical diagnostics.
10.Piflufolastat F 18 for PSMA PET imaging in prostate cancer
Jing-Tian SHI ; Ting YANG ; Chao-Yang CHEN ; Ran WEI ; Xuan-Ling ZHANG ; Xiao-Juan HU ; Ying ZHOU
The Chinese Journal of Clinical Pharmacology 2024;40(12):1835-1838
On May 27,2021,the U.S.Food and Drug Administration(FDA)officially approved Lantheus'PYLARIFY?(Piflufolastat F 18,18 F-labeled imaging agent),which can be used for positron emission computed tomography(PET)of prostate-specific membrane antigen(PSMA)-positive lesions in prostate cancer patients to accurately identify prostate cancer with suspected metastasis or recurrence.Piflufolastat F 18 is approved by FDA for two indications.The first is the initial staging for suspected metastatic lesions in men with newly diagnosed prostate cancer.The second is restaging,with the goal of identifying lesions in the setting of biochem ical recurrence.

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