The purpose of this study is to determine whether dietary cholic acid therapy benefits people with Smith-Lemli-Opitz syndrome (SLOS) by leading to an increase in plasma cholesterol and reduction in harmful cholesterol precursors. SLOS participants will be treated with dietary cholic acid for 8 weeks and plasma cholesterol and cholesterol precursor metabolites will be measured.

开始日期2021-03-27

申办/合作机构

University of Nebraska

[+3]

CTIS2024-518652-23-00

/ Completed临床4期IIT

Long-term safety study of personalized cholic acid treatment in patients with bile acid synthesis defects

开始日期2020-06-01

申办/合作机构-

NL-OMON25657

/ RecruitingN/A

Long-term safety study of personalized cholic acid treatment in patients with bile acid synthesis defects

开始日期2020-05-18

申办/合作机构-

100 项与胆酸相关的临床结果

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100 项与胆酸相关的转化医学

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100 项与胆酸相关的专利（医药）

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8,625

项与胆酸相关的文献（医药）

2027-01-01·JOURNAL OF NUTRITIONAL BIOCHEMISTRY

Dietary iron imbalance disrupts enterohepatic homeostasis via the gut microbiota–bile acid–FXR signaling axis

Article

作者: Wu, Tong ; Zhou, He ; Da, Yu ; Liang, Jie ; Fan, Zhenzhen ; Shi, Yanting ; Wang, Fang ; He, Jindan ; Lin, Junchao ; Zhang, Jiaqi

Dietary iron deficiency and excess may both disturb enterohepatic homeostasis, yet their differential effects on organ-specific iron distribution and the microbiota-bile acid-FXR signaling axis remain unclear. Here, we integrated multi-cohort cross-sectional analyses with mouse experiments, cell co-culture assays, organoid models, metabolomics, and fecal microbiota transplantation experiments. In population analyses, higher dietary iron, rather than serum iron, was associated with diarrhea, fatty liver, and liver fibrosis. In mice, iron deficiency induced widespread reductions in tissue iron content, whereas iron excess caused marked iron accumulation in the small intestine, colon, liver, spleen, kidney, and skeleton. Both iron-deficient and iron-excess diets impaired colonic barrier homeostasis, as indicated by reduced goblet cell abundance, lower Muc2 expression, disrupted tight junctions, and increased intestinal permeability. However, iron excess caused more profound gut microbiota dysbiosis, perturbed bile acid-related bacterial taxa, depleted primary bile acids, suppressed intestinal and hepatic farnesoid X receptor (FXR) signaling, and exacerbated hepatic steatosis, fibrosis, inflammation, and dysfunction. Importantly, chenodeoxycholic acid, cholic acid, and fecal microbiota transplantation partially restored bile acid-FXR signaling, improved barrier integrity, and alleviated liver injury under iron-excess conditions. Collectively, dietary iron imbalance disrupts enterohepatic homeostasis via the gut microbiota-bile acid-FXR signaling axis, with iron excess emerging as the predominant driver of bile acid dysregulation, impaired FXR signaling, and gut-liver injury. These findings identify dietary iron as an important determinant of enterohepatic homeostasis and support bile acid- and microbiota-targeted strategies for iron-related metabolic disorders.

2026-12-31·Virulence

Article

作者: Ouyang, Zirou ; Yang, Huimin ; Qin, Pu ; Niu, Yanan ; Qiang, Cuixin ; Wen, Baojiang ; Zhao, Jianhong ; Wang, Weigang ; Zhao, Min ; Zhao, Jiafeng ; Yang, Jing

Clostridioides difficile infection (CDI) is a major cause of antibiotic-associated diarrhea, with frequent recurrences closely linked to antibiotic-induced dysbiosis of the gut microbiota and bile acid metabolism. Parabacteroides distasonis, a potential probiotic capable of converting primary to secondary bile acids, has shown therapeutic promise in several metabolic and inflammatory diseases. This study evaluated the preventive and therapeutic effects of P. distasonis against CDI and explored the underlying mechanisms. We characterized the probiotic properties of four P. distasonis strains and investigated the inhibitory activity of strain 1190003 against C. difficile, as well as its protective and therapeutic efficacy in mouse models. Gut microbiota structure and bile acid metabolic profiles were analyzed by integrating 16S rRNA gene sequencing and metabolomics. The four P. distasonis strains exhibited strong acid and bile salt tolerance as well as auto-aggregation ability. Supernatants from P. distasonis co-cultured with cholic acid (4 mM and 8 mM) significantly inhibited C. difficile growth, toxin expression and spore formation, with deoxycholic acid identified as the key inhibitory metabolite. Both live P. distasonis and its culture supernatant alleviated disease severity in CDI mouse models and ameliorated gut microbiota dysbiosis. Notably, the relative abundance of Parabacteroides goldsteinii was increased following supernatant treatment. Furthermore, intervention with either live P. distasonis or its supernatant elevated the level of hyodeoxycholic acid. In summary, P. distasonis acts as a potential probiotic that alleviates CDI by ameliorating gut microbiota dysbiosis and remodeling bile acid metabolism. These findings provide experimental evidence for its use as a microbiota-based therapeutic strategy.

2026-12-01·JOURNAL OF MOLECULAR HISTOLOGY

Ebselen attenuates selected cardiac and vascular alterations in rats exposed to a cholesterol/cholic acid diet

Article

作者: Vardi, Nigar ; Parlakpinar, Hakan ; Ermis, Necip ; Yildiz, Azibe ; Soysal, Kubra ; Turkoz, Yusuf ; Demez, Sumeyye ; Tasdemir, Seda ; Ozhan, Onural

Hypercholesterolemia (HCT) is a major risk factor for cardiovascular diseases and promotes myocardial and vascular injury primarily through oxidative stress (OS)-mediated mechanisms. Ebselen (EBS), a glutathione peroxidase-mimetic antioxidant, has been associated with attenuation of tissue injury in several experimental OS-related models; however, its short-term effects on selected outcomes in HCT-associated cardiovascular damage remain unclear. Twenty-four male Wistar albino rats were randomly assigned to three groups: Control, cholesterol/cholic-acid diet (HCT), and the same diet treated with EBS (HCT+EBS). Rats in the HCT-designated groups received a diet containing 2% cholesterol and 0.5% cholic acid for six weeks; serum lipids were not measured. EBS (10 mg/kg/day, intraperitoneal) was administered during the final week. Hemodynamic and electrocardiographic parameters, serum injury biomarkers, myocardial oxidative-stress parameters, and cardiac and descending-aortic histopathology were assessed. Relative to Control, the cholesterol/cholic-acid diet group designated HCT showed higher myocardial lipid peroxidation, lower superoxide dismutase (SOD) activity, higher serum myoglobin and lactate dehydrogenase, and more severe cardiac and aortic histopathological alterations. Compared directly with HCT, HCT+EBS showed lower myocardial malondialdehyde, higher SOD activity, lower injury biomarkers, and less severe histopathological alterations. Because serum lipids were not measured, these differences cannot be separated from a possible EBS-associated difference in lipid profile. EBS administration also induced a marked hypotensive response: systolic blood pressure was significantly lower in HCT+EBS than in both HCT and Control. Short-term EBS administration was associated with selected biochemical and histopathological differences in rats exposed to a cholesterol/cholic-acid diet. However, the lipid phenotype was not confirmed, and a possible EBS-associated difference in serum lipids cannot be excluded. Together with the absence of an EBS-only control group, this prevents attribution of the findings specifically to direct antioxidant actions of EBS or interpretation as therapeutic efficacy.

195

项与胆酸相关的新闻（医药）

2026-10-26

·BioSpace

Mirum Pharmaceuticals Announces Positive Results from Cohort 2 of the Pivotal PROGRESS Study of ATEBRIOZ™ (zilurgisertib) in Pediatric Patients Aged 6 to <12 Years with Fibrodysplasia Ossificans Progressiva

- Zilurgisertib reduced total HO lesion volume and improved clinician-assessed joint mobility; no zilurgisertib-treated patients developed new HO lesions - Mirum plans to submit an sNDA in H1 2027 to expand th e ATEBRIOZ indication to pediatric patients aged 6 to <12 years - Safety and efficacy findings consistent with PROGRESS Cohort 1; no new safety signals FOSTER CITY, Calif.--(BUSINESS WIRE)--Mirum Pharmaceuticals, Inc. (NASDAQ: MIRM), a leading rare disease company, today announced positive results from Cohort 2 of the pivotal PROGRESS study of ATEBRIOZ ™ (zilurgisertib), an oral activin receptor-like kinase 2 (ALK2) inhibitor, in pediatric patients aged 6 to <12 years with fibrodysplasia ossificans progressiva (FOP). At Week 24, zilurgisertib reduced total heterotopic ossification (HO) lesion volume and improved clinician-assessed joint mobility. No zilurgisertib-treated patients developed new HO lesions. The results build on data from PROGRESS Cohort 1, which supported the U.S. Food and Drug Administration (FDA) approval of ATEBRIOZ in September 2026 for adult and pediatric patients aged 12 years and older with FOP. Mirum plans a supplemental New Drug Application (sNDA) submission in H1 2027 to expand the indication to pediatric patients aged 6 to <12 years. “These results represent an important advance for younger children living with FOP,” said Rob Myers, M.D., Chief Medical Officer of Mirum. “The reduction in total HO volume and absence of new lesions seen with zilurgisertib build on the positive results previously observed in adolescents and adults in Cohort 1 of PROGRESS. We look forward to discussing these findings with the FDA to expand access to ATEBRIOZ for younger children living with this debilitating disease.” “FOP is a devastating genetic disease that leads to progressive loss of mobility, independence, quality of life, and shortened lifespan,” said Frederick S. Kaplan, M.D., Isaac and Rose Nassau Professor of Orthopaedic Molecular Medicine at the University of Pennsylvania Perelman School of Medicine. “Childhood is a critical window in FOP, so a therapy targeting the underlying cause of the disease is especially important for this young age group. Results such as these offer hope that we may be able to slow disease progression in children before its most serious consequences arise.” PROGRESS Cohort 2 Results Cohort 2 of the PROGRESS study enrolled 25 pediatric patients aged 6 to <12 years with FOP. Patients were randomized 1:1 to receive once-daily oral zilurgisertib 75 mg (n=12) or placebo (n=13) during a 24-week double-blind period, followed by an open-label extension. The mean age at baseline was 8.3 years, with a mean of 5.0 years since initial diagnosis. All patients completed the double-blind period and entered the open-label extension. At Week 24, mean total HO lesion volume decreased by 8.09 cm³ in patients receiving zilurgisertib, compared with an increase of 9.14 cm³ in patients receiving placebo (nominal p=0.003). No zilurgisertib-treated patients developed new HO lesions. Improvements in clinician-assessed joint mobility were also observed. Joint mobility was assessed using the Cumulative Analogue Joint Involvement Scale (CAJIS), a validated measure of physical function in FOP scored from 0 (no impairment) to 30 (maximum impairment). At Week 24, CAJIS scores improved by a mean of 0.80 points in patients receiving zilurgisertib, compared with 0.09 points in patients receiving placebo. In untreated patients with FOP, CAJIS scores are reported to worsen by approximately 0.5 points per year. 1 Key efficacy data from Cohort 2 are shown below in the Efficacy Results table. Efficacy Results Endpoint Zilurgisertib 75 mg QD Placebo Key Finding Mean (SD) change from baseline in total HO lesion volume, cm³ -8.09 (15.58) n=12 9.14 (11.66) n=13 Treatment difference a of -14.0 cm³ favoring zilurgisertib (nominal p=0.003). b Patients with new HO lesions, n (%) 0 (0) n=12 1 (7.7) n=13 No new HO lesions with zilurgisertib. Mean (SD) change from baseline in CAJIS score -0.80 (1.1) n=10 -0.09 (1.0) n=11 Improvement in clinician-assessed joint mobility with zilurgisertib. a Hodges-Lehmann estimate of treatment difference b p-value calculated using a Wilcoxon rank-sum test Zilurgisertib was generally well tolerated, with a safety pro with that observed in PROGRESS Cohort 1. No new safety signals were identified. No patients discontinued treatment during the double-blind period, and no zilurgisertib-treated patients experienced growth plate closure. Additional safety findings included: Most adverse events were mild or moderate in severity. Serious adverse events and Grade ≥3 adverse events were infrequent and occurred less frequently in patients receiving zilurgisertib than in those receiving placebo. Adverse events occurring in more than 10% of zilurgisertib-treated patients and at a higher frequency than with placebo included arthralgia, cough, pyrexia, and vomiting (3 subjects each), and alopecia, epistaxis, fall, iron deficiency, and nasopharyngitis (2 subjects each). ATEBRIOZ is approved by the U.S. FDA to reduce the volume of total new HO in adult and pediatric patients aged 12 years and older with FOP. Total new HO lesion volume includes expansion of baseline HO lesion burden as well as any new discrete HO that developed during the 24-week double-blind period. Full Cohort 2 results will be presented at an upcoming medical meeting. Cohort 3 of the PROGRESS study, which is evaluating zilurgisertib in pediatric patients aged 2 to <12 years, is currently enrolling. ATEBRIOZ is available in the U.S. through Mirum Access Plus (MAP), a patient support program designed to help patients, families, and healthcare providers navigate treatment access. MAP provides insurance coverage and access support, financial assistance for eligible patients, personalized patient support, and educational resources for patients and caregivers. Eligible patients can pay as little as $0 per month. To learn more, call 855-MRM-4YOU (1-855-676-4968) or visit atebrioz.com . About ATEBRIOZ™ (zilurgisertib) tablets ATEBRIOZ™ (zilurgisertib) tablets are a once-daily oral activin receptor-like kinase 2 (ALK2) inhibitor approved by the U.S. Food and Drug Administration (FDA) to reduce the volume of total new heterotopic ossification in adult and pediatric patients aged 12 years and older with Fibrodysplasia Ossificans Progressiva (FOP). In people living with FOP, pathogenic variants in the ACVR1 gene result in abnormal activation of ALK2, leading to the formation of bone in muscles, tendons, ligaments and other soft tissues through a process known as heterotopic ossification (HO). For more information, please visit atebrioz.com . Mirum Pharmaceuticals, Inc. licensed zilurgisertib from Incyte for worldwide development and commercialization. IMPORTANT SAFETY INFORMATION ATEBRIOZ is contraindicated in pregnancy. ATEBRIOZ can cause fetal harm based on data from animal studies. Patients of reproductive potential should use effective contraception and should immediately discontinue ATEBRIOZ and contact their healthcare provider if pregnancy occurs. US Prescribing Information About the PROGRESS Study PROGRESS is a global, randomized, double-blind, placebo-controlled Phase 2 study evaluating the efficacy and safety of zilurgisertib in patients with fibrodysplasia ossificans progressiva (FOP). PROGRESS Cohort 1 enrolled 63 patients 12 years of age and older who were randomized 1:1 to receive zilurgisertib 100 mg once daily or placebo during a 24-week double-blind treatment period, followed by an open-label extension. Results from Cohort 1 have been reported through Week 48. The PROGRESS study is also evaluating the safety and efficacy of zilurgisertib in younger patients with FOP. Cohort 2 enrolled 25 patients aged 6 to <12 years who were randomized 1:1 to receive zilurgisertib 75 mg once daily or placebo during a 24-week double-blind treatment period, followed by an open-label extension. Cohort 3, which is evaluating zilurgisertib in patients aged 2 to <12 years, is currently enrolling. About Fibrodysplasia Ossificans Progressiva (FOP) Fibrodysplasia ossificans progressiva (FOP) is an ultra-rare, progressive genetic disease affecting approximately 300 people in the United States and 900 worldwide. FOP is characterized by heterotopic ossification (HO), a process in which bone forms in muscles, tendons, ligaments and other soft tissues. Symptoms typically become apparent in early childhood, and the number and volume of HO lesions increase over time, progressively restricting movement and limiting mobility, daily function, and independence. About Mirum Pharmaceuticals Mirum Pharmaceuticals (NASDAQ: MIRM) is a leading rare disease company with a global footprint of approved products and a broad pipeline of investigational medicines. Purpose-built to bring forward breakthrough medicines for people with overlooked conditions, Mirum focuses on rare liver and rare genetic diseases, where it has built deep expertise and strong connections to patient communities. The company’s commercial portfolio includes LIVMARLI® (maralixibat) for Alagille syndrome (ALGS) and progressive familial intrahepatic cholestasis (PFIC), ATEBRIOZ™ (zilurgisertib) for fibrodysplasia ossificans progressiva (FOP), CHOLBAM® (cholic acid) for bile-acid synthesis disorders and CTEXLI® (chenodiol) for cerebrotendinous xanthomatosis (CTX). Mirum’s clinical-stage pipeline includes volixibat, an IBAT inhibitor in late-stage development for primary sclerosing cholangitis (PSC) and primary biliary cholangitis (PBC), brelovitug, a fully human monoclonal antibody in late-stage development for chronic hepatitis delta virus (HDV) and MRM-3379, a PDE4D inhibitor being evaluated for Fragile X syndrome (FXS). Mirum’s success is driven by a team dedicated to advancing high impact medicines through strategic development, disciplined execution and purposeful collaboration across the rare disease ecosystem. Learn more at and follow Mirum on Facebook , LinkedIn , Instagram and X. Mirum Forward-Looking Statements Statements contained in this press release regarding matters that are not historical facts are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements include statements regarding, among other things, expectations regarding the timing, enrollment or results of clinical trials for zilurgisertib, the potential benefit of zilurgisertib in real world settings versus clinical trial settings, including the potential to slow disease progression in children before FOP’s most serious consequences arise, the timing of regulatory engagement with the FDA and any resulting submission of an sNDA for ages 6 to <12, the importance of ATEBRIOZ™ as an additional therapy for the treatment of FOP, the potential presentation of zilurgisertib clinical results at an upcoming medical meeting, cost to patients and methods of availability. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Words such as “plan,” “may,” “expected,” “will,” “could,” “would,” “guidance,” “potential,” “continue” and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based upon Mirum’s current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, risks and uncertainties associated with the development of investigational medicines generally, including the failure of future studies to generate the same or similar data as prior studies, risks inherent in the regulatory submission process in the U.S., risks and uncertainties associated with Mirum’s business in general, the impact of geopolitical and macroeconomic events, and the other risks described in Mirum’s Quarterly Report for the quarter ended June 30, 2026, filed with the Securities and Exchange Commission on August 5, 2026, and subsequent filings with the Securities and Exchange Commission, which are available at . All forward-looking statements contained in this press release speak only as of the date on which they were made and are based on management’s assumptions and estimates as of such date. Mirum undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law. Mirum and the Mirum logo are trademarks of Mirum Pharmaceuticals, Inc. References 1 Pignolo RJ, et al. Orphanet J Rare Dis. 2019;14:113. Contacts Mirum Investor Contact: Andrew McKibben ir@mirumpharma.com Mirum Media Contact: Meredith Kiernan media@mirumpharma.com

临床结果上市批准临床2期

2026-10-22

·中草药杂志社

中药质量标志物（Q-Marker）10年回顾与展望

自2016年刘昌孝院士[1-3]创造性地提出中药质量标志物（quality marker，Q-Marker）的概念，迄今已经历时10年。Q-Marker概念与核心理论的提出，密切了中药有效性、物质基础与质量控制标志性成分之间的关联，着眼于全过程的物质基础特有、差异、动态变化和质量传递性、溯源性，解决了中药质量研究工作碎片化的现象，开创了中药质量研究新模式。有利于建立中药全程质量控制与质量溯源体系，促进中药质量的科学监管。10年来，围绕中药Q-Marker主题，业界开展了广泛深入的研究，在理论阐释、模式创新、关键技术和产业应用等方面取得了长足进展。徐园园等[4]基于中国知网与Web of Science数据库，对2016年5月1日至2023年5月31日发表的1 030篇中英文Q-Marker文献进行系统的科学计量分析，结果显示该领域研究总体呈快速增长态势，并已形成以中药质量控制、药效物质基础、炮制机制、中药药理与安全性评价、中药资源种植为核心的5大研究方向。截至2026年8月30日，公开发表的中药Q-Marker研究论文总数已达1 500余篇。本文围绕Q-Marker的理论创建、研究路径方法、研究实践进展、产业化应用及新技术前沿5个方面进行系统论述，并对当前研究面临的挑战和未来发展方向进行展望，为Q-Marker的深入研究与推广应用提供参考。 1 Q-Marker理论创建及其科学内涵阐释 1.1 Q-Marker新概念及其核心理论构建 2016年刘昌孝院士等[1]基于中药特点和中药质量控制需求，首次创造性提出中药Q-Marker的概念，并明确中药Q-Marker核心内容有效、特有、传递与溯源、可测和处方配伍的“五要素”。其后，经过系统的整理，撰写出版了系统的理论专著《中药质量标志物理论与实践》[5]。形成了中药Q-Marker的理论体系。中药Q-Marker新概念与核心理论针对中药复杂体系特点和全产业链系统质控要求，以有效性、特有性、可测性、传递与溯源及配伍环境“五要素”为核心内容，有利于建立与中药临床效用密切关联、特有专属、整体评价和面向中药全产业链的质量标准和质量控制体系，并为行业监管提供科学、有效的“监管工具”，对于提高我国中药质量标准和质量控制水平具有重大的现实意义[6]。 1.2 Q-Marker的确定原则与研究路径为了进一步明确中药Q-Marker的确定原则和研究路径，Zhang等[7]总结归纳了中药Q-Marker的概念、核心理论与研究方法，提出中药Q-Marker确定的“五原则”，以“有效”和“特有”反映质量评价体系与有效性关联和专属性特点；以“传递与溯源”贯穿药材-中间产品-成药的全程质量控制；以“可测”呈现中药质量属性的全貌；以“配伍环境”回归中药临床作用的客观实质。基于“五原则”的Q-Marker研究，既反映了与有效性和安全性的关联关系，又体现中药成分的专属性、差异性特征，特别是基于方-证对应的配伍环境使质量研究回归到中医药理论的本质内涵，有利于规范中药质量研究和标准建立，克服现有质量标准多种不足，提高质量一致性与可控性。在此基础上，进一步就有效性、特有性、传递与溯源[8-10]等进行了分别论述，为中药Q-Marker研究明确了确定原则、研究方法和技术路径。需要说明的是，“五要素”与“五原则”既相互关联又各有侧重：刘昌孝院士等[1]提出的“五要素”（有效、特有、传递与溯源、可测、处方配伍）是中药Q-Marker的核心内涵与判定标准，回答“什么是Q-Marker”的问题；张铁军等[8]在系统总结基础上归纳提炼的“五原则”则是面向研究实践的确定原则与技术路径，回答“如何发现和确定Q-Marker”的问题。二者一脉相承，“五原则”是“五要素”的继承、细化与发展，将理论内涵转化为可操作的研究范式，共同构成中药Q-Marker从概念提出到研究实践的完整理论框架。 1.3 中药Q-Marker研究方法探讨中药Q-Marker既是一个整合的理论也是开放的理论。国内学者对中药Q-Marker的研究方法进行了广泛而深入的探讨，形成了多元化的理论体系与技术路径。张铁军等[11]提出基于“性-效-物三元论”的有效性表达视角和研究模式。白钢等[12]结合药效学评价和近红外技术，提出从化学标志物-Q-Marker-近红外快检的研究路径。王丹妮等[13]提出采用“蛛网膜式”进行中药Q-Marker的筛选。刘晓娜等[14]提出多源信息融合的中药Q-Marker与质量评价研究模式。唐于平等[15]以“有效性为核心、可测性与特有性为条件，复方配伍为背景”的遴选思路，构建“分层三维整合技术”量化评价，并基于层次分析-熵权法综合评分排序筛选Q-Marker的新方法。许海玉等[16]提出基于整合药理学的中药Q-Marker发现与确证思路。侯小涛等[17]提出基于反向功效差异性特点的中药Q-Marker研究策略。江振作等[18]提出“药材基原-物质基础-Q-Marker-质控方法”层级递进的中药质量标准新模式。 1.4 中药Q-Marker研究指南在中药Q-Marker的核心理论指导下，基于中药Q-Marker确定的“五原则”，在广泛深入研究的基础上，2025年中华中医药学会正式发布《中药质量标志物研究技术指南》（T/CACM 1638-2025）团体标准[19]。该指南系统明确了中药Q-Marker发现和确定的原则、研究路径、技术方法，及基于Q-Marker的中药质量评价方法和质量标准研究内容。指南适用于中药材、饮片、提取物、配方颗粒、煎液、中成药的Q-Marker研究，为构建中药Q-Marker研究的科学模式、推动中药全程质量溯源体系建设和产业监管提供了规范性指导。自2016年中药Q-Marker新概念提出以来，业内进行了广泛的理论探索、方法创新和实验研究，虽然已有中药Q-Marker理论、技术方法和确定原则的文献报道[6,20]，但一直以来，Q-Marker研究仍属于开放式的探索研究，该标准的发布实施标志着Q-Marker研究从学术探索迈向标准化、规范化新阶段，实现理论依据向标准化的跨越，成为行业Q-Marker研究的参照和指南，进一步规范中药质量研究、指导全程质量控制体系建立并提供科学监管的手段和重要工具，对提升中药质量控制水平、推动中药产业高质量发展具有重要意义。 2 Q-Marker的研究实践已发表的中药Q-Marker的文章涉及理论、技术方法及中药材、饮片和上市后中成药的Q-Marker研究，包括专论、Q-Marker预测性综述和研究性论文。 2.1 药材与饮片Q-Marker研究在药材和饮片的Q-Marker研究方面，据不完全统计涉及470味药材，以基于“五原则”的Q-Marker预测分析的综述性论文占绝大多数。目前已对当归、党参、大黄、延胡索、五味子、何首乌、泽泻、茯苓、蒲黄、麻黄、桔梗、川芎、三七等200余种进行了深入研究，代表性20种药材和饮片研究见表1。张铁军等[21]通过HPLC-Q/TOF-MS技术，对醋延胡索药材进行化学物质组辨识，鉴定出28个生物碱类化合物。进一步通过分析延胡索生物碱类成分生源途径，确定延胡索乙素、延胡索甲素等为特异性成分。结合药效学实验，整体动物模型显示延胡索提取物可延长热板法舔足潜伏期、减少醋酸扭体次数、抑制痛经模型扭体反应；离体实验显示其可降低缩宫素引起的子宫收缩。网络药理学分析表明，延胡索乙素、巴马汀、海罂粟碱、原阿片碱可作用于9个蛋白靶点及9条信号通路，主要与中枢镇痛相关。药性研究中，延胡索乙素与原阿片碱与苦味、辛味受体对接亲和力强，并激活5-羟色胺1A受体、μ-阿片受体、β2肾上腺素受体等G-蛋白偶联受体（G-protein-coupled receptor，GPCR）。药动学研究显示，7个原型成分可穿过血脑屏障进入脑组织，发挥中枢镇痛作用。综合上述研究结果确定延胡索乙素、延胡索甲素、黄连碱、巴马汀、去氢延胡索甲素、D-四氢药根碱及原阿片碱7个生物碱为Q-Marker。多项研究采用UPLC-Q/TOF-MS结合多元统计方法对莪术炮制前后的化学物质组进行表征辨识及差异分析；通过多种病证动物模型和细胞模型对生、醋莪术饮片的药效进行比较研究，结合代谢组学、信号通路等深入探索了莪术醋制增效的作用机制，并运用双位点微透析、荧光成像示踪技术比较主要活性成分的药动学差异；进一步综合考虑成分的可测性，最终确定以莪术二酮、莪术醇、吉马酮、呋喃二烯和β-榄香烯作为生、醋莪术饮片的Q-Marker[21-23]。 2.2 中药复方Q-Marker研究中药复方的有效性是基于方-证对应的配伍环境，强调要使质量研究回归到中医药理论的本质内涵，“配伍环境”是中药Q-Marker研究的核心内容之一。中药复方制剂的Q-Marker研究能够完整体现Q-Marker“五要素”的全部内容。迄今，据不完全统计，已对疏风解毒胶囊、元胡止痛滴丸、麝香保心丸等180余个中药复方进行了研究，代表性的20个中药大品种见表2。张铁军等[42]对中药大品种疏风解毒胶囊的Q-Marker进行了系统研究。首先采用液-质联用技术对疏风解毒胶囊处方药材、制剂及血中移行成分进行系统辨识，从8味药材中鉴定了174个化学成分、制剂中94个、血浆中46个化合物，阐明了“药材-制剂-体内”全过程的化学物质组传递与溯源关系，并基于生源途径分析了化学成分的特有性。继而综合运用整体动物模型、谱效分析、网络药理学、基因组学、蛋白质组学、仿生模型及功能受体结合等方法，结合“靶点钓取-结合验证-机制解析”策略，运用热蛋白质组学、细胞热漂移测定、微量热泳动、分子对接与分子动力学模拟、氨基酸定点突变等前沿技术[43]，系统阐明其药效物质基础、作用机制及配伍规律，最终确定虎杖苷、大黄素、连翘酯苷A、戟叶马鞭草苷、马鞭草苷、毛蕊花糖苷及甘草酸等可作为疏风解毒胶囊的Q-Marker。中药大品种麝香保心丸基于整体观的研究思路，整合血清药物化学、代谢组学、网络药理学和多组分配伍研究等方法确定Q-Marker[44]。首先通过HPLC-DAD-MS鉴定出47个化学成分（人参皂苷类、蟾蜍甾烯类、胆酸类等），GC-MS鉴定49个挥发性成分，大鼠口服血清药物化学发现35个入血成分（27个原型＋8个代谢物）。在人脐静脉内皮细胞模型上评价了26个入血成分的促血管新生活性，确定了7个活性组分配伍成分，分别为肉桂酸、麝香酮、蟾毒灵、人参皂苷Rb1、人参皂苷Re、胆酸、冰片。进一步在急性心肌梗死大鼠模型上证实，该7个组分配伍的疗效优于任一单体，体现了多成分协同作用。最终上述7个成分被确定为麝香保心丸的Q-Marker。对中药大品种痹祺胶囊进行系统的Q-Marker研究[45]。首先采用UPLC-Q/TOF-MS技术对10味原料药材进行系统辨识，共鉴定262个化学成分、制剂中280个成分，并借助血清药物化学方法在大鼠血浆中鉴定了81个外源性化合物（含59个原型成分与22个代谢产物），明确了“药材成分组-制剂成分组-血行成分组”的传递与溯源规律，并基于生源途径分析了化学成分的特有性。基于网络药理学预测了39个代表性化合物的124个作用靶点、71条作用通路；通过小鼠单核巨噬细胞白血病RAW264.7炎症模型、风湿关节炎成纤维样滑膜细胞增殖模型与破骨细胞分化模型筛选出19个抗炎药效物质基础；进一步采用GPCR与酶活检测实验，评价19个主要单体成分对类风湿性关节炎关键靶点的调节作用，明确其作用机制；拆方研究将痹祺胶囊分为益气养血、活血通络、除湿止痛3组，通过功能模型验证了3组协同增效的配伍合理性。最终确定士的宁、马钱子碱、甘草苷、丹酚酸B、甘草酸、隐丹参酮与丹参酮IIA共7个成分为Q-Marker，最后，建立了基于Q-Marker的“点-线-面-体”多元质控方法，将原标准仅控制2个生物碱提升为全息指纹图谱与多指标一测多评相结合的全面质量控制体系。 2.3 药材与复方制剂Q-Marker研究共性规律及核心差异 “五原则”是中药Q-Marker确定的核心原则，但针对药材和复方制剂的Q-Marker研究具有各自的倾向和差异。药材研究更注重多元功效有效性的“完整表达”，在可测性方面应针对不同功效分别建立多个质量指标的质量标准；在特有性方面，更宜基于植物亲缘学、化学成分的生源途径、化学成分的环境事变依据、采收加工的物候期依据、化学成分的显微组织化学依据、炮制加工的成分转化依据等综合判断，因缺失配伍环境和制备工艺环节，不能完全体现“五原则”的完整性。复方制剂能完整体现“五原则”的全部内涵，但在有效性的表达上更应体现基于临床方-证/病对应的临床治疗环境下有效性的“针对表达”；在传递与溯源方面，能体现“植物-药材-饮片-制剂-体内”中药全生命周期的完整过程；在可测性方面，更宜基于关键质量属性建立“点-线-面-体”结合的多元质量控制方法和整体质量标准。 3 Q-Marker的应用 3.1 科学研究中药Q-Marker自提出以来，得到了国家自然科学基金委员会、科技部、国家药品监督管理局等政府机构的高度重视和持续资助，已发展为中药质量研究领域的前沿和焦点方向。Q-Marker概念的提出开创了中药质量研究新模式，引领了我国中药质量研究的创新发展，相关研究成果在学术界、产业界和药品监管部门产生了广泛而深远的影响[62-63]。 2018年国家自然科学基金（Natural Science Foundation of China，NSFC）专门设立课题对Q-Marker研究进行研讨，并给予了系统性支持，中药Q-Marker研究被列入NSFC重点项目资助方向，当年获批3项重点项目：“基于中医方证代谢组学的关黄柏质量标志物发现”“基于成分构成-靶标网络-生物效应多维整合的中药质量标志物发现与确认”和“基于临床真实世界证据的中药质量标志物的发现与确认”[64]。2017—2023年，NSFC累计资助Q-Marker相关面上项目、青年基金、地区科学基金和国际合作项目39个，包括黄芪、泽泻、枳壳、附子、地黄、重楼、滇黄精、荷叶、莲子心、莲须、大黄炭、栀子、当归、绞股蓝、巴戟天、降香、茯苓、炙甘草、香加皮、金钗石斛、金银花、北葶苈子、银柴胡、黑骨藤、扁担藤、翼首草、血筒等中药材，及双黄连制剂、血脂灵片、金莲清热颗粒、鸡骨草胶囊、宣肺败毒颗粒、复方三七血伤宁、冠心舒通方、木丹颗粒、平痔胶囊等中成药品种。2024—2025年，持续支持中药Q-Marker研究10余项，涉及五加皮、重楼、红禾麻、大风藤参等药材和枣仁安神颗粒、宁泌泰胶囊、羌活胜湿汤等复方制剂及创新方法学研究。中药Q-Marker研究产生了广泛的国内国际学术影响，在Phytomedicine期刊出版了2期Q-Marker专刊、《中草药》期刊出版了2期Q-Marker专刊，在《中草药》《药学学报》《中国现代中药》等分别出版了Q-Marker专栏文章。目前，除中国外，美国、英国、法国、印度、韩国等国家的学者也在开展相关研究[65-66]，表明Q-Marker概念已获得国际学术界的认同。“中药Q-Marker创新理论、关键技术与转化应用”获得天津市科技进步一等奖。17篇Q-Marker相关论文获得中国科协百篇优秀论文和F5000优秀论文（表3）。 3.2 产业应用 3.2.1 标准提升以中药Q-Marker统领，对现行的中药材、饮片和中成药的质量标准进行提升研究，有利于深化质量控制指标与临床功效的相关性、质量评价的整体性及质量标准的专属性。张铁军团队在Q-Marker理论指导下，对三七、延胡索、白芍等药材开展了系统的质量标准提升研究。以三七为例，在系统辨识化学物质组及传递规律的基础上，通过体内外模型阐明其活血、止血、消肿定痛等多重功效的药效物质基础，确定三七素、三七皂苷R1、人参皂苷Rg1、Re、Rb1、Rg2、Rd等为Q-Marker，建立了基于Q-Marker的指纹图谱及“一测多评”多指标含量测定方法，实现三七粉质量标准的全面提升[29,67]。多项研究以金银花和当归为对象，基于核因子-κB抑制和Ca2+拮抗等生物效价筛选确立抗炎、血管舒张Q-Marker，并利用近红外光谱技术实现“量效转换”的药材质量快速整合评价，为药材质量标准提升提供了新范式[68-70]。熊亮等[71]以益母草和赶黄草为例，从药效物质基础、成分专属性、化学结构与生物活性、可测性及指纹图谱5个方面系统研究其Q-Marker，为药材标准提升提供了示范。中成药大品种多为中药复方制剂，其质量属性最能完整体现中药Q-Marker“五原则”的特点。通过大品种二次开发系统研究，聚焦凝练Q-Marker，进一步提升质量标准，是质量标准提升的可行路径。在中药大品种二次开发研究的基础上，对疏风解毒胶囊、痹祺胶囊、元胡止痛滴丸、注射用益气复脉（冻干）、六经头痛片、活血止痛胶囊等中药大品种质量标准进行了全面提升。对疏风解毒胶囊[72-73]，通过化学物质组与抗炎药效相关性研究，确定10个成分为主要抗炎药效物质基础，建立了6个指标成分定量测定方法和指纹图谱控制方法。对元胡止痛滴丸[11,74]，基于“性-效-物”三元论研究模式，从味觉、嗅觉和功能属性表达角度筛选药味物质基础，从整体动物模型、离体器官、网络药理学及代谢组学等层次和方法确定胡索甲素、延胡索乙素、原阿片碱、欧前胡素和异欧前胡素为Q-Marker，相比《中国药典》规定的欧前胡素和延胡索乙素含量测定指标，赋予了新的科学内涵并丰富了质量控制体系。对六经头痛片[75-76]，通过药材、成品及口服入血成分的辨识和表征阐释化学物质组，经GPCR结合实验及网络药理学分析筛选明确主要药效物质基础，通过拆方研究与同类中药及化学药比较阐释组方特点和配伍规律；建立HPLC指纹图谱共有模式及3′-羟基葛根素、葛根素、大豆苷、特女贞苷、大豆苷元5个指标成分的定量测定方法，对原有质量标准进行了全面提升。 3.2.2 追溯系统建设中药Q-Marker研究强调质量的传递与溯源，刘昌孝等[77-78]从中药资源、产业的角度，提出基于Q-Marker的中药质量追溯系统建设，通过从药材源头到成品全过程的质量、标准和控制研究，建立从药材、饮片、中间产物到制剂的符合中药特点的质量标准体系，构建全程可溯源的质量控制方案，是保证中药质量和产业全程控制的关键，有利于促进中药标准化[12]。近年来，数字化和智能化技术的飞速发展为中药质量追溯系统的升级提供了新路径。安徽省药监局联合亳州市政府启动的中药饮片信息化追溯系统建设，通过“一物一码、物码同追”实现了中药饮片来源可查、去向可追、质量可控、责任可究[79]。国药太极等企业通过建设中药材良好农业规范追溯展示平台，利用5G、大数据、区块链、物联网、人工智能等信息技术，实现了从田间到成品的全过程质量追溯。天津药品监管信息化平台升级“风险预警”“数据分析”“追溯查询”三大模块，实现了基于Q-Marker的量质传递数据实时监测与风险预警[80]。这些实践表明，将Q-Marker的量质传递数据与信息化追溯平台深度融合，是中药产业现代化的重要方向。在中药智能制造背景下，张磊等[81]首次提出了基于数据驱动的中药制药过程Q-Marker监测方法，将Q-Marker概念从离线质量评价拓展至在线生产过程监测。该方法包含3个关键步骤：中药Q-Marker监测指标识别、制药工业质量数据挖掘及过程分析技术（process analytical technology，PAT）体系建立。依托PAT平台，集成传感器、统计分析和模式识别技术，可实现关键工艺参数与质量属性的实时关联分析。注射用益气复脉（冻干）是Q-Marker研究和全程质量控制体系构建的典型范例，通过系统的基础研究筛选确定中药Q-Marker，提升质量标准，建立从药材、加工、提取、制造、贮藏、药物警戒等全过程，建立符合6个管理规范的全产业链中药质量控制体系，实现了从田间到病床的完整闭环[82]。依托国家中药标准化项目，疏风解毒胶囊开展了系统标准化建设，建立基于Q-Marker的多指标成分定量与指纹图谱相结合的质量控制方法，并构建“药材-饮片-中间体-制剂”全过程质量控制体系。“疏风解毒胶囊二次开发的系统研究及应用”获安徽省科技进步一等奖。 3.3 中药质量监管中药质量涉及产业链一系列环节，各个生产环节的生产质量管理规范与中药制剂的质量关系极大，监管的重点在中药药材来源监管、饮片加工炮制规范、成药制剂工艺过程风险控制的科学性和适用性。基于“质量源于设计”的理念，开展中药材、中药饮片和中成药的Q-Marker研究，明确药物关键质量属性在产业链不同环节之间的量质传递过程，对关键环节和风险控制点加强控制，制定科学规范的中药质量标准体系、评价指导原则及技术指南，能够有效地推进中药产业的科学监管与健康发展[83]。白钢等[84]提出了基于监管科学的中药质量评价方法的整合研究思路，强调应将Q-Marker作为中药质量评价的核心指标，建立以Q-Marker为抓手的全过程质量监管体系。张铁军团队承担完成国家药品监督管理局“中国药品监管科学行动计划”项目，以“疏风解毒胶囊”“注射用益气复脉（冻干）”“痹祺胶囊”3个代表性中药大品种为示范，建立了中药Q-Marker的发现模式、确定原则与技术方法，构建了基于Q-Marker的全生命周期质量控制体系。为中药行业科学监管提供了关键技术手段，有力支撑了中药监管科学体系建设。 4 新技术与新方法在Q-Marker研究中的应用随着分析化学、数据科学和生命科学技术的飞速发展，一系列新技术和新方法在Q-Marker的发现、验证和应用中展现出巨大潜力。本文重点介绍近年来在该领域应用的新技术方向。 4.1 质谱成像（mass spectrometry imaging，MSI）与仿生传感技术 MSI技术特别是基质辅助激光解吸电离质谱成像（matrix-assisted laser desorption/ionization mass spectrometry imaging，MALDI-MSI），能够在组织切片水平上同时获取多种化学成分的空间分布信息，为中药Q-Marker的发现提供了全新的空间维度视角[85]。Deng等[86]开展了基于MALDI-MSI技术解析不同采收期亳白芍的空间代谢组学研究，揭示了芍药苷等活性成分在药材不同组织中的空间分布规律及其随采收期的动态变化，为白芍Q-Marker的确定提供了空间分布证据。《中国药典》2025年版已首次收载质谱成像技术作为分析方法[87]，标志着该技术已从科学研究走向标准应用。仿生传感技术在中药“药性”辨识方面展现出独特优势。电子舌和电子鼻技术通过模拟人体味觉和嗅觉感知系统，可以客观量化中药的“五味”和“滋味、气味”特征[88]。延胡索的Q-Marker研究中就采用了电子舌/电子鼻仿生技术，结合苦味受体hTAS2R10和辛味受体嗅觉受体家族7亚家族D成员4的分子对接筛选，确定辛苦药味的“真实五味”，进一步通过离体、细胞及GPCR生物效应模型方法，确定“功效五味”，成功将“药性”物质基础与Q-Marker相关联[21]。炒紫苏子研究中，Heracles II电子鼻技术分析筛选出52个变量重要性投影＞1差异挥发性成分，为阐释炒紫苏子“焦香气”的物质基础提供了科学数据[89]。这些技术将传统中医药“感官评价”转化为可量化的科学指标，为基于“药性”的Q-Marker研究开辟了新途径。 4.2 多组学整合与中医方证代谢组学中药Q-Marker的研究正在从单一分析技术向多组学整合的方向发展。系统融合代谢组学、蛋白质组学、转录组学及网络药理学等多维数据，可从“化学物质-生物靶点-药效效应-临床应用”全链条阐明Q-Marker科学内涵[90]。中医方证代谢组学是整合血清药物化学与代谢组学的研究策略，其核心思路是在“方证对应”并显效的前提下，通过代谢组学技术从内源性小分子代谢层面表征证候代谢轮廓、发现生物标记物，采用血清药物化学方法阐明体内显效成分，进而将体内显效成分暴露水平与病证生物标记物表达水平进行关联分析，筛选高度相关的效应关联成分，并确认成分所属药材（体现君、臣、佐、使），结合可测性、特有性最终确定Q-Marker[54]。刘绍博等[91]基于方证代谢组学联合靶向蛋白分析策略，在风寒湿痹证骨关节病大鼠模型中进行痹痛宁胶囊Q-Marker的深入挖掘，通过非靶向代谢组学提取病症生物标记物、联合生物信息学方法提取核心蛋白靶点，进行体内直接作用物质的分子对接分析，最终确定异甘草苷、香橙素、5,3',4'-三羟基黄酮为痹痛宁胶囊Q-Marker。茵陈蒿汤治疗阳黄证[92]和脑灵片治疗失眠症[93]的药效物质基础研究，进一步拓展了该策略的应用范围。方证代谢组学已从单纯的代谢组学整合发展为“代谢物-蛋白-基因”多层面、多维度的系统生物学研究范式[94]，为Q-Marker的“有效性”提供了更加全面的科学证据。 4.3 中药相态分析与多维确证策略近年来，中药相态研究成为了新的研究热点，为中药Q-Marker研究提供了新的认识视角和研究空间。徐少华等[95]提出“自上而下-自下而上相结合”的双向融合策略：（1）自上而下的发现策略，依照《中国药典》制备汤剂，通过监测煎煮过程中粒径、分散系数、化学成分的变化及芘荧光探针测定临界聚集浓度确定工艺；采用差速离心、膜透析、尺寸排阻、超滤拆分相态，以“分辨率-回收率-活性保留率-污染程度”四维评价规范操作；经细胞/动物药效筛选最佳活性相态后，运用聚山梨酯20、NaCl、尿素及超声反向干预，联合X射线单晶衍射、冷冻电镜、分子动力学模拟解析聚集体结构与靶向机制。（2）自下而上的确证策略，以标准品模拟合成自组装聚集体，经形态与理化表征后通过药理与分子生物学验证不同功效Q-Marker。基于相态研究开展Q-Marker研究，可将关联特定功效的活性超分子聚集体确立为多维功效Q-Marker，破解“一物多效”中药Q-Marker确证难题，推动质控从“成分还原论”向“结构-功能一体化”创新转变，为构建符合中药特点的质量评价新范式提供科学依据。 4.4 人工智能与机器学习人工智能与机器学习技术在高维数据处理、非线性建模与模式识别方面展现独特优势，为Q-Marker发现、体内过程预测及质量智能化管理提供了新的技术手段[96]。中药成分复杂，单味药或复方常含数百至数千种化学成分，传统实验筛选效率低、成本高。机器学习通过构建预测模型可从海量化合物库中快速筛选活性成分，支持向量机（support vector machine，SVM）、随机森林（random forest，RF）等模型在活性成分初筛、靶点预测及吸收、分布、代谢、排泄性质评价中表现优异[97]。Zhang等[98]利用UPLC-Q-TOF-MS/MS结合正交偏最小二乘判别分析及SVM、RF等模型，从贯叶连翘中鉴定出候选Q-Marker。Ma等[99]将薄层色谱图像与LC-HRMS多维指纹融合，以RF筛选特征并结合信息熵权重评价，从复方阿胶浆中确定党参炔苷为成品特异标志物、人参皂苷Rf为中间体特异标志物。Bai等[100]提出智能质量评价策略，以UPLC-Q/TOF结合偏最小二乘判别分析筛选化学标志物并确证为Q-Marker后，经近红外光谱快速定量，再通过人工神经网络、遗传算法-支持向量回归预测药材整体功效，并集成电子舌、电子鼻等智能感知技术与远程通信构建质量管理系统。 5 结语与展望经过10年发展，围绕“有效、特有、传递与溯源、可测、处方配伍”五原则的Q-Marker研究路径方法已形成系统的理论与技术体系，在药材、饮片及复方研究中积累了大量成功案例，为中药质量标准提升提供了坚实的科学基础。在应用层面，Q-Marker正成为中药质量追溯系统建设与产业监管科学的核心抓手，《中药质量标志物研究技术指南》（T/CACM 1638-2025）团体标准的发布标志着该领域从学术探索迈向标准化新阶段。尽管Q-Marker研究已取得显著进展，但仍面临若干挑战。（1）迄今大多数研究还停留在候选Q-Marker筛选与预测层面，Q-Marker的实验研究和确证尚有不足；（2）有效性的研究多针对少数指标，有效性的完整表达研究较少；（3）药材Q-Marker研究较多，基于“五原则”的中药复方制剂Q-Marker研究较少；（4）基于Q-Marker的质量标准提升研究较少；（5）基于Q-Marker的全程质量控制体系建设较少。基于“方证对应”临床治疗环境的有效性表达，及面向标准提升与全程质量控制体系转化的行业应用，将成为未来研究与应用的关键与重点。具体将体现在以下5个方面。 5.1 基于有效性表达的深刻认识，进一步深化Q-Marker研究 “有效性”是Q-Marker“五原则”的“重中之重”，因此，有效性的认识和表达是Q-Marker研究的试金石。在中医药理论当中，“药性”与“药效”（功效）均是中医药理论的核心概念，是中药特有的功效属性，是从不同侧面、不同角度对中药治疗疾病性能的客观描述，反映中药有效性的本质特征，并作为临证治法、遣药组方的重要依据。“药味（性）”和“药效”体现中药的“物质基础”作用人体疾病主体的不同层面、不同方式的生物效应表达形式，因此，“性-效-物”三元论是中药有效性认识的基本视角。更重要的是，有效性的研究既要体现中药材的多元药效的“完整表达”，又要根据“方-证对应”的临床配伍环境的“针对表达”。在研究方法和技术路径方面，国内学者提供了广泛的探索和研究范例，如方证代谢组学、态靶理论等。进一步广泛和深入的探索研究对于完善Q-Marker理论，深化和关联与临床的安全性、有效性具有重要的意义。 5.2 现代科学技术的广泛应用，为Q-Marker研究提供更多方法和手段近年来，现代科学技术突飞猛进的发展，并不断向中药学领域应用，极大的推动了中药现代化进程。Q-Marker创新理论的发展迫切要现代科学技术的支持和推动，多组学技术基于系统论的理论内核，与中医药理论的整体观和复杂体系特点深度契合。在系统-系统层面，中药化学物质复杂体系与生物效应表达的复杂体系是Q-Marker研究的基本层面，关联化学物质-生物效应-生信分析，进行整合分析是Q-Marker发现的重要手段和技术路径；另一方面，化学成分-效应靶点的确认是Q-Marker的最终节点，因此，要素-要素还原论的研究方法是Q-Marker研究的必要手段，网络药理学方法、靶点垂钓等化学生物学方法进一步广泛用于Q-Marker研究；现代分析技术为中药的物质组的解析、体内过程分析及质量控制方法提供了重要的保障，将不断推动Q-Marker深入研究、标准提升和质量控制体系的构建。 5.3 大数据与人工智能为Q-Marker研究创造新的研究模式大数据和人工智能使中药研究发生了深刻的变化，如本草基因组学、泛靶标数据库、人工智能、跨域大模型、区块链等颠覆了中药研究的传统模式。Q-Marker“五要素”中涉及的有效性、物质基础及其量值传递、特有性及生源途径均需要传统数据和现代研究证据的支持。数据挖掘、智能算法将极大的推动中药Q-Marker的研究和发展，一方面多学科整合研究和数据分析为基于特有性的Q-Marker的发现提供更多生源学和亲缘学证据，同时另一方面泛靶标数据库和人工智能“干湿结合”研究为中药药效物质基础解析及基于有效性的Q-Marker的发现提供更多的技术方法和研究路径，并进一步从定性向定量层次的跨越。 5.4 Q-Marker进一步向行业应用与产业下沉 2025年2月《中药质量标志研究技术指南》的发布，进一步规范了Q-Marker研究的技术方法，实现了Q-Marker研究的关键节点。未来，Q-Marker研究将进一步向质量标准提升过渡，Q-Marker将成为质量标准提升的重要内容和质量控制指标，推动质量标准与临床安全有效性的深度关联，提高质量标准的专属性和实际应用价值；在此基础上，充分体现Q-Marker传递与溯源的特点，以提升的质量标准为尺子，建立多环节、多节点的全程质量控制体系，实现“Q-Marker确定-质量标准提升-全程质量控制体系建立”的转化应用，推动中药产业的标准化进程，提高中药产业的质量控制水平。 5.5 成为监管科学的重要抓手和工具中药质量成因和影响因素复杂，质量评价方法体系不完整、质量控制手段缺乏和质量参差不一是中药产业的难点和痛点，也是制约中药科学监管的关键瓶颈。既往质量标准单一和少数指标与有效性关联不强，不能满足对中药安全有效性的评价与控制，也难以作为科学监管的有效手段和工具。Q-Marker基于中药理论体系特点和中药产业特点，以“有效”“特有”“传递与溯源”“配伍环境”“可测”五要素为核心内容，使中药的质量评价和质量控制实现了实质性飞跃，Q-Marker列入了国家药品监督管理局监管行动计划第二批课题并通过验收，实现了从科学研究-产业应用-行业监管的转化应用。通过研究-产业-医疗-监管多环节的联动，将进一步推动Q-Marker的深入研究、标准提升、产业应用和科学监管。提高我国中药质量标准和质量控制水平，促进中药产业健康发展。利益冲突所有作者均声明不存在利益冲突参考文献（略）来源：张铁军，白钢，陈常青，许浚．中药质量标志物（Q-Marker）10年回顾与展望 [J]. 中草药, 2026, 57(20): 7947-7960.

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2026-10-22

·BioSpace

Mirum Pharmaceuticals to Participate in Upcoming November 2026 Investor Conferences

FOSTER CITY, Calif.--(BUSINESS WIRE)--Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM), a leading rare disease company, today announced that it will participate in the following upcoming investor conferences: Wolfe Research Healthcare Conference 2026 : Tuesday, November 10, 2026 Company fireside chat starting at 8:00 a.m. ET Stifel 2026 Healthcare Conference : Wednesday, November 11, 2026 Company fireside chat starting at 1:20 p.m. ET 2026 Jefferies Global Healthcare Conference : Tuesday, November 17, 2026 Company presentation starting at 12:30 p.m. GMT The presentation and fireside chats will be webcast live and can be accessed by visiting the Investors section of Mirum’s corporate website. The archived webcasts will be available for replay. About Mirum Pharmaceuticals Mirum Pharmaceuticals (NASDAQ: MIRM) is a leading rare disease company with a global footprint of approved products and a broad pipeline of investigational medicines. Purpose-built to bring forward breakthrough medicines for people with overlooked conditions, Mirum focuses on rare liver and rare genetic diseases, where it has built deep expertise and strong connections to patient communities. The company’s commercial portfolio includes LIVMARLI® (maralixibat) for Alagille syndrome (ALGS) and progressive familial intrahepatic cholestasis (PFIC), ATEBRIOZ™ (zilurgisertib) for fibrodysplasia ossificans progressiva (FOP), CHOLBAM® (cholic acid) for bile-acid synthesis disorders and CTEXLI® (chenodiol) for cerebrotendinous xanthomatosis (CTX). Mirum’s clinical-stage pipeline includes volixibat, an IBAT inhibitor in late-stage development for primary sclerosing cholangitis (PSC) and primary biliary cholangitis (PBC), brelovitug, a fully human monoclonal antibody in late-stage development for chronic hepatitis delta virus (HDV) and MRM-3379, a PDE4D inhibitor being evaluated for Fragile X syndrome (FXS). Mirum’s success is driven by a team dedicated to advancing high impact medicines through strategic development, disciplined execution and purposeful collaboration across the rare disease ecosystem. Learn more at and follow Mirum on Facebook , LinkedIn , Instagram and X . Contacts Investor Contact: Andrew McKibben ir@mirumpharma.com Media Contact: Meredith Kiernan media@mirumpharma.com

上市批准

100 项与胆酸相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D10699	胆酸	A05AA03	DB02659

研发状态

批准上市

10 条最早获批的记录,

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适应症	国家/地区	公司	日期
局部肥胖	韩国	Medytox Inc.	2025-09-19
胆汁酸和胆固醇代谢紊乱	日本	Recmed	2023-03-27
胆汁酸合成障碍	美国	Mirum Pharmaceuticals, Inc.	2015-03-17
肝病	美国	Mirum Pharmaceuticals, Inc.	2015-03-17
过氧物酶体病	美国	Mirum Pharmaceuticals, Inc.	2015-03-17
脂肪泻	美国	Mirum Pharmaceuticals, Inc.	2015-03-17
Zellweger综合征	美国	Mirum Pharmaceuticals, Inc.	2015-03-17
先天性胆汁酸合成障碍1型	英国	Laboratoires C.T.R.S. SAS	2013-09-12
先天性胆汁酸合成障碍2型	英国	Laboratoires C.T.R.S. SAS	2013-09-12
先天性胆汁酸合成障碍	欧盟	Theravia SAS	2013-09-12
先天性胆汁酸合成障碍	冰岛	Theravia SAS	2013-09-12
先天性胆汁酸合成障碍	列支敦士登	Theravia SAS	2013-09-12
先天性胆汁酸合成障碍	挪威	Theravia SAS	2013-09-12

未上市

10 条进展最快的记录,

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适应症	最高研发状态	国家/地区	公司	日期
肾上腺脑白质营养不良	临床3期	美国	Mirum Pharmaceuticals, Inc.	1992-01-01
胆汁淤积	临床3期	美国	Mirum Pharmaceuticals, Inc.	1992-01-01
婴儿反流疾病	临床3期	美国	Mirum Pharmaceuticals, Inc.	1992-01-01

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临床结果

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT03720990 (CTgov)	临床1/2期	Smith-Lemli-Opitz综合征	12	Cholic Acid	網網淵鏇製淵鏇顧鹽淵(夢鏇夢淵範獵製簾範選) = 齋鏇糧鬱顧壓鑰願鏇鹹鹽襯遞選選鑰鬱鬱糧醖 (顧窪醖製糧築獵繭艱蓋, 22.4) 更多	-	2023-12-13
NCT01438411 (CTgov)	临床3期	胆汁酸合成障碍 \| 先天性胆汁酸合成障碍	53	Cholic Acid	鹹淵網壓衊鹹糧鏇獵艱 = 艱壓壓遞積壓鬱蓋膚簾顧觸鑰襯遞憲衊夢構衊 (範獵夢簾糧糧艱願範範, 醖觸餘夢膚鏇夢艱獵鏇 ~ 衊獵艱鏇壓鑰憲鹹遞壓) 更多	-	2020-08-21
NCT00007020 (CTgov)	临床3期	婴儿反流疾病 \| 胆汁淤积 \| 肾上腺脑白质营养不良 ... 更多	85	Cholic Acids	衊鑰積鑰壓積鹽鏇繭觸 = 顧願繭積網範選膚觸鏇鹹範膚壓醖網遞窪醖觸 (衊鑰鏇遞簾鹽窪鏇壓餘, 鹽憲築壓廠積鹹壓蓋鑰 ~ 夢築範構壓鬱顧範憲簾) 更多	-	2020-07-15
NCT00457639 (CTgov)	临床2期	脂肪营养不良 \| 脂肪肝	18	Cholic Acid (Cholic Acid)	鑰繭簾廠鬱製顧築獵淵(餘餘蓋齋夢顧顧繭廠觸) = 齋構築衊簾網齋鹹鹽繭廠襯夢觸醖鹽夢鑰餘淵 (簾繭繭簾鹽築選蓋製積, 範獵願廠憲簾獵廠觸築 ~ 糧選壓淵築獵製築鏇醖) 更多	-	2019-01-15
NCT00457639 (CTgov)	临床2期	脂肪营养不良 \| 脂肪肝	18	Placebo (Placebo)	鑰繭簾廠鬱製顧築獵淵(餘餘蓋齋夢顧顧繭廠觸) = 鏇蓋築齋簾夢繭鹽積艱廠襯夢觸醖鹽夢鑰餘淵 (簾繭繭簾鹽築選蓋製積, 鏇鬱艱餘蓋齋憲蓋糧鬱 ~ 網襯選齋襯範蓋鹽鹹顧) 更多	-	2019-01-15
NCT01115582 (CTgov)	临床3期	先天性胆汁酸合成障碍	16	Cholic Acid	鏇糧觸簾遞鹹繭鑰網簾(餘夢鹽壓構築壓繭遞醖) = 鏇範積鬱憲壓窪築鹽醖繭廠衊築顧夢觸齋壓淵 (鏇壓憲齋膚築齋繭鑰憲, 21.9) 更多	-	2016-12-09
Pubmed \| Eur J Endocrinol	临床2期	脂肪肝	18	Cholic acid	窪鑰獵餘蓋鬱襯艱艱範(鹽積鹽淵構製簾獵糧遞) = 積膚簾夢艱醖顧鹽獵遞選窪壓淵窪鏇願遞夢顧 (窪築鑰夢鑰獵糧選憲壓, 9.4 ~ 19.0) 更多	不佳	2013-05-01
Pubmed \| Eur J Endocrinol	临床2期	脂肪肝	18	Placebo	窪鑰獵餘蓋鬱襯艱艱範(鹽積鹽淵構製簾獵糧遞) = 廠蓋鏇膚膚築齋鑰獵膚選窪壓淵窪鏇願遞夢顧 (窪築鑰夢鑰獵糧選憲壓, 10.5 ~ 26.5) 更多	不佳	2013-05-01