爱塔乐伦(Ataluren) - 药物靶点：DAG1_在研适应症：杜氏肌营养不良症_专利_临床

概要

基本信息

药物类型

小分子化药

别名

3-[5-(2-fluorophenyl)-1,2,4-oxadiazol-3-yl]benzoic acid、Ataluren (USAN/INN)、阿他卢仑

+ [5]

靶点

DAG1

作用方式

刺激剂

作用机制

肌营养不良蛋白聚糖（Dystroglycan）刺激剂

治疗领域

神经系统疾病遗传病与畸形皮肤和肌肉骨骼疾病+ [1]

在研适应症

杜氏肌营养不良症

非在研适应症

无虹膜贝克型肌营养不良囊性纤维化+ [7]

原研机构

PTC Therapeutics, Inc.

在研机构

PTC Therapeutics International Ltd.

PTC Therapeutics, Inc.ROVI Pharma Industrial Services SA

+ [3]

非在研机构

Frosst Iberica

权益机构-

最高研发阶段批准上市

首次获批日期

欧盟 (2014-07-31),

杜氏肌营养不良症

最高研发阶段(中国)申请上市

特殊审评快速通道 (美国)、孤儿药 (美国)、孤儿药 (欧盟)、孤儿药 (韩国)、孤儿药 (澳大利亚)、优先审评 (中国)

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结构/序列

分子式C15H9FN2O3

InChIKeyOOUGLTULBSNHNF-UHFFFAOYSA-N

CAS号775304-57-9

关联

37

项与爱塔乐伦相关的临床试验

JPRN-jRCT2071220020

/ Completed临床1期IIT

A Single-Dose Study to Assess the Food Effect on the Pharmacokinetics and Tolerability of Ataluren (PTC124) in Healthy Japanese Subjects

开始日期2022-06-24

申办/合作机构-

NCT04336826

/ Completed临床2期

An Open-Label Study Evaluating the Safety and Pharmacokinetics of Ataluren in Children From ≥6 Months to <2 Years of Age With Nonsense Mutation Duchenne Muscular Dystrophy

This study is designed to evaluate safety, tolerability, and pharmacokinetics (PK) in male children with nmDMD aged ≥6 months to <2 years treated daily for 24 weeks with orally administered ataluren 10, 10, and 20 milligrams/kilogram (mg/kg) (morning, mid-day, and evening dose, respectively).

开始日期2021-12-29

申办/合作机构

PTC Therapeutics, Inc.

CTR20211795

/ Active, not recruiting临床3期

一项 ATALUREN (PTC124) 在无义突变型杜兴氏肌营养不良患者中的长期安全性、疗效和耐受性的开放性研究

本研究的目标在于评估长期 Ataluren 10 mg/kg 、10 mg/kg 和 20 mg/kg 给药在完成 III 期研究 (PTC124-GD-041-DMD )后希望继续接受治疗的 nmDMD 受试者中的安全性、疗效和耐受性。将根据所有不良事件 (AE)、实验室检查异常、生命体征、心电图 (ECG)、超声心动图和体格检查结果的类型、频率、严重程度、时间以及与 Ataluren 的关系来描述 Ataluren 的安全性特征。Ataluren 的疗效将根据上肢功能 (PUL)、DMD 上肢患者报告结局指标 (PROM)、肺量测定、行走能力丧失 (LoA)和 6 分钟步行试验 (6MWT)较基线的变化来评估。

开始日期2021-09-14

申办/合作机构

ROVI Pharma Industrial Services SA

[+2]

100 项与爱塔乐伦相关的临床结果

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100 项与爱塔乐伦相关的转化医学

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100 项与爱塔乐伦相关的专利（医药）

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283

项与爱塔乐伦相关的文献（医药）

2026-03-01·SPECTROCHIMICA ACTA PART A-MOLECULAR AND BIOMOLECULAR SPECTROSCOPY

Unraveling the interaction mechanism between the genetic disease drug Ataluren and eukaryotic codon recognition factor 1/ human serum albumin through spectroscopic and nuclear magnetic resonance approaches

Article

作者: Tian, Hong ; Duan, Ran ; Guo, Hanlin ; Yu, Juping ; Peng, Zhengxiong ; Yu, Xinyu ; Liu, Wei

PTC124 (Ataluren) is a small molecule drug for the treatment of genetic diseases that has been shown to promote early termination codon read-through. In this study, the binding model of PTC124 to human serum albumin and eukaryotic stop codon recognition factor (eRF1) was established in vitro. In the cell model, the read-through effect of PTC124 on the stop codon was confirmed. Fluorescence analysis showed that PTC124 could spontaneously bind eRF1 and HSA, the binding constants were 3.132 ± 0.643 × 10⁴ and 2.963 ± 0.218 × 10⁵ L/mol (298 K), respectively. Thermodynamic analysis indicated that the binding to eRF1 was driven mainly by hydrophobic interactions, hydrogen bonding, and van der Waals forces, whereas the binding to HSA was driven primarily by hydrogen bonding and van der Waals forces. Structural analysis revealed perturbations in the secondary structure of both HSA and eRF1 induced by PTC124, while the overall native folds of the proteins were maintained. In addition, a binding model of the interaction between PTC124 and eRF1/HSA was established by molecular simulation and STD-NMR. The binding model described in this paper explains the interaction mechanism between PTC124 and its utility and transport targets at the molecular level, which is helpful for the design and synthesis of new molecules related to PTC124 structure and based on its mechanism of action.

2026-03-01·Molecular Therapy-Nucleic Acids

Beyond the stop: Oxadiazole TRIDs restore LRBA protein expression in nonsense-driven primary immunodeficiency

Article

作者: Lentini, Laura ; Pibiri, Ivana ; Moutschen, Michel ; Meier-Menches, Samuel M ; Pace, Andrea ; Ricci, Davide ; Zito, Antonino ; Vitale, Emanuele ; Borutzki, Yasmin ; Fiduccia, Ignazio ; Colige, Alain ; Bileck, Andrea ; Varrica, Riccardo ; Marino, Sefora

Nonsense mutations are among the genetic causes of LRBA (lipopolysaccharide-responsive beige-like anchor) deficiency, a rare autosomal-recessive immunodeficiency disorder. These mutations introduce premature stop codons, leading to the loss of LRBA protein expression. Following the recent market withdrawal of ataluren, the only approved translational readthrough-inducing drug (TRID), there is an urgent need for alternative therapeutic options. In this study, we investigated the efficacy of three 1,2,4-oxadiazole-based TRIDs-NV848, NV914, and NV930-using primary fibroblasts from a patient homozygous for the R1683X nonsense mutation. All compounds restored full-length LRBA protein with correct cytoplasmic localization, as confirmed by western blot and immunofluorescence, outperforming ataluren in readthrough efficiency. NV848 exhibited the strongest activity and uniquely increased LRBA mRNA levels, suggesting transcript stabilization. In contrast, NV930 and NV914 induced readthrough without stabilizing mRNA. Global proteomic profiling revealed minimal off-target effects for NV848, limited protein modulation by NV914, and widespread variations of 828 proteins by NV930, affecting pathways related to vesicular transport and mRNA splicing. However, network analysis revealed poor connectivity among differentially expressed proteins, with LRBA unrelated to any regulated cluster. These findings highlight the reported molecules as promising candidates for precision therapy in LRBA deficiency and shed light on the broader cellular impact of TRIDs.

2026-01-01·Advanced Science

TREM2‐Mediated Cholesterol Efflux in Macrophages Inhibits Anti‐Tumor Immunity via Limitation of CD4 ⁺ T and NK Cells

Article

作者: Aitian Li ; Qitai Zhao ; Li Yang ; Qingyang Lei ; Weina Yu ; Yunhan Wang ; Yi Zhang ; Shasha Liu ; Tian Wang ; Xinxin Wang

Abstract:

Tumor‐associated macrophages (TAMs) predominantly exert functions that facilitate tumor progression. Triggering receptor expressed on myeloid cell 2 (TREM2) is expressed in TAMs, playing a crucial role in mediating the immunosuppressive function of TAMs. The mechanisms by which TREM2 + TAMs promote tumor growth and inhibit anti‐tumor immunity remain unclear. Through single‐cell sequencing of tumor tissues derived from wild‐type and Trem2 knockout mice bearing subcutaneous lung cancer, it is found that TREM2 deletion hindered tumor growth, with a notable increase in and improved functionality of CD4 + T and natural killer (NK) cells in the tumor microenvironment. TREM2 deficiency led to ATP‐binding cassette transporter A1 (ABCA1) downregulation, causing cholesterol accumulation in TAMs and promoting a pro‐inflammatory phenotype. This results in increased chemokine (C‐X3‐C motif) ligand 1 (CX3CL1) secretion of macrophages, recruiting more CD4 + T and NK cells to the tumor site, enhancing the anti‐tumor response. After screening food and drug administration (FDA)‐approved drugs, bortezomib and ataluren are found to effectively inhibit TREM2 expression in TAMs, indicating a potential therapeutic strategy against TREM2. This study elucidates the mechanism by which TREM2 shapes the immunosuppressive microenvironment and promotes tumorigenesis, highlighting TREM2 as a target for cancer immunotherapy.

112

项与爱塔乐伦相关的新闻（医药）

2026-02-16

·FiercePharma

PTC shuts down FDA approval bid for troubled Duchenne med Translarna

Translarna has a checkered past with the FDA and, despite scoring a thumbs-up in Europe back in 2014, recently met a major marketing setback there, as well. With the writing apparently on the wall, PTC Therapeutics has called off its latest bid for FDA approval of its Duchenne muscular dystrophy (DMD) drug Translarna. Late Thursday, PTC revealed that it withdrew its FDA application for Translarna in nonsense mutation DMD after receiving feedback on its filing from the regulator. "FDA shared that based on its review to date, the data in the [New Drug Application] submission are unlikely to meet the Agency's threshold of substantial evidence of effectiveness to support approval of Translarna," Matthew Klein, M.D., PTC's chief executive, explained in a statement. PTC is "disappointed that FDA approval cannot be achieved," Klein said, citing the decades of development work that have gone into the company's protein restoration therapy. In a letter to the U.S. DMD community, PTC cited unresolvable "differences in data interpretation" with the FDA, Pharmaphorum reports. Translarna has a checkered past with the FDA and, despite scoring a thumbs-up in Europe back in 2014, recently met a major marketing setback there, as well.Stateside, the FDA initially declined to review PTC's Translarna application in 2016, telling the company its application was incomplete. About a year and a half later, the FDA formally rejected the drug, citing the need for PTC to run an additional trial "at a minimum" to better validate Translarna's effectiveness. The FDA also flagged other "nonclinical and [chemistry, manufacturing and controls] matters" in the approval bid.PTC announced it would make another attempt at U.S. approval in early 2024, pointing to "recent feedback" from the FDA. Now, that effort is ending without success.The drug has also suffered recent regulatory setbacks in the EU and, despite some pushback from European officials, the European Medicines Agency's human medicines committee ultimately decided not to renew the drug's conditional marketing authorization in late 2024. The European Commission, which has final say on approvals in the bloc, made the same call last March. European regulators blessed the med with a conditional nod in 2014 on results from a phase 2b study that favored Translarna over placebo on a six-minute walk test after 48 weeks of treatment. But that study and a subsequent trial did not produce statistically significant results on the metric.Back in the U.S., PTC also faced FDA friction around its Friedreich's ataxia candidate vatiquinone last year when the regulator denied approval of the candidate over a lack of "substantial evidence of efficacy" in the company's filing. Translarna, which goes by the generic moniker ataluren, is a protein restoration therapy that is designed to allow the formation of a functioning protein in patients with genetic disorders caused by nonsense mutations. PTC describes that type of mutation as an alteration in the genetic code that prematurely halts the synthesis of an essential protein. In the case of DMD, that protein would be dystrophin.

临床结果上市批准

2026-02-16

·Pharmaceutical Technology

PTC pulls Translarna NDA after unfavourable FDA feedback

It is currently unclear if PTC plans to re-file for Translarna’s US approval in DMD. Image credit: Michael Vi via ShutterStock.com. PTC Therapeutics has chosen to withdraw its resubmitted new drug application (NDA) for Duchenne muscular dystrophy (DMD) therapy, Translarna (ataluren), marking another significant setback in the drug’s US development programme. This call comes after the US Food and Drug Administration (FDA) voiced concerns over the drug’s efficacy in nonsense mutation DMD. According to PTC’s CEO, Matthew Klein, the regulator shared that based on its review thus far, the data used to support the application is “unlikely to meet the agency’s threshold of substantial evidence of effectiveness” to support Translarna’s approval. For this reason, the rare disease specialist has chosen to pull the NDA submitted to the FDA for Translarna in DMD, marking the third unsuccessful attempt by PTC to secure Translarna’s approval in the US. The company did not immediately respond to Pharmaceutical Technology’s request when asked if it plans to re-file for the drug’s approval with the FDA. The FDA previously refused to review the dystrophin protein restoration therapy in 2016, citing the incomplete nature of its associated NDA as the primary reason. On PTC’s second attempt to commercialise the drug in 2017, which followed a successful appeal of the FDA’s 2016 decision, the drug was once again rejected. This time, the decision was on the grounds that at least one additional study would be necessary to prove the drug’s efficacy in DMD. Translarna’s future in DMD market under question Alongside its troubles in getting the US regulatory greenlight, Translarna has also been subject to regulatory woes in the European market. GlobalData Strategic Intelligence US Tariffs are shifting - will you react or anticipate? Don’t let policy changes catch you off guard. Stay proactive with real-time data and expert analysis. By GlobalData Learn more about Strategic Intelligence While the drug first gained conditional marketing authorisation from the European Medicines Agency (EMA) in 2014, the agency later issued a negative opinion on renewing Translarna’s approval in 2024, following the review by the European Commission (EC) in late 2023. Now, the drug is only available to countries choosing to leverage Articles 117(3) and 5(1) of the EU Directive 2001/83, according to a 2025 statement from PTC. However, the drug remains approved for use in the UK. The drug is also available through the National Health Service (NHS). GlobalData, parent company of Pharmaceutical Technology , forecasts that Translarna will generate $73m in global sales during 2031 – down significantly from its $356m 2023 sales peak. Translarna is not the only DMD drug to receive a negative opinion from the FDA in recent months, as Capricor Therapeutics received a complete response letter (CRL) for its cell therapy, deramiocel, back in September 2025. In January 2026, the company issued an update, stating that it is currently compiling a clinical study report and supporting data in a bid to resubmit its application for deramiocel in DMD. Meanwhile, Sarepta’s approved DMD gene therapy, Elevidys (delandistrogene moxeparvovec), was also temporarily pulled from the US market following two non-ambulatory patient deaths after treatment. The FDA later reinstated Elevidys on the market following a review.

申请上市上市批准细胞疗法基因疗法

2026-02-15

·微信

长达 12 年的“西西弗斯”式悲剧：PTC 官宣撤回 Translarna 上市申请！面对 FDA 的死刑判决，DMD 患者最后的希望碎了？

在罕见病研发的江湖里，杜氏肌营养不良（DMD）一直是一座极难攻克的堡垒。 2026 年 2 月 13 日，PTC Therapeutics 悄然发布了一则令人心碎的公告：正式撤回其明星药物 Translarna (ataluren) 在美国的上市申请（NDA）。这意味着，这款在欧洲已经销售了 12 年、在 FDA 门外徘徊了三次的药物，彻底关上了通往美国市场的大门。从 2014 年的意气风发，到 2026 年的败走麦城，Translarna 的陨落不仅是一家药企的战略失误，更是一场关于“临床有效性”与“监管红线”的残酷博弈。一、最后的通牒：FDA 没给任何机会周四晚间，PTC 首席执行官 Matthew Klein 博士在声明中表现出了极度的失望。他透露，FDA 在最近的反馈中明确表示：基于目前的审查情况，Translarna 的数据“极不可能”满足证明其有效性的实质性证据门槛。这并非 FDA 第一次“拒绝”它。回顾 Translarna 的入美之路，堪称一部灾难片： 2016 年： FDA 甚至拒绝审评 PTC 的申请，理由是“资料不全”。 2017 年： FDA 正式拒绝批准，要求 PTC 至少再跑一次临床试验来证明有效性。 2024 年初：借着所谓的“监管风向好转”，PTC 试图发动第三次冲击。然而，2026 年初的 FDA 比以往任何时候都更加务实。在给 DMD 患者群体的公开信中，PTC 承认与 FDA 之间存在“无法调和的数据解读差异”。二、欧美的“双重绞杀”：从先行者变成流浪者 Translarna 曾有过辉煌的过去。2014 年，它凭借二期临床数据获得了欧盟的“附条件上市许可”，成为全球首款针对无义突变型 DMD 的蛋白质恢复疗法。然而，谎言终会被戳破。根据协议，PTC 必须在后续试验中证明药物能显著改善患者的 6 分钟步行测试（6MWT）。可悲的是，随后的多项大规模三期试验中，Translarna 在统计学上均未达到显著差异。 2024 年末，欧洲药品管理局（EMA）旗下的 CHMP 委员会终于失去了耐心，决定不再续展其上市许可。尽管 PTC 拼命游说，但在 2025 年 3 月，欧盟委员会正式维持了“撤市”决定。随着美国申请的撤回，Translarna 在全球主流市场已经面临“全线清场”的终局。三、深度解析：为什么“无义突变”疗法如此难做？ Translarna 的设计思路极具魅力：它旨在让核糖体“跳过”基因代码中的错误终止信号，从而合成出功能完整的抗肌萎缩蛋白（Dystrophin）。理论很丰满：如果成功，它将从源头修复遗传缺陷。现实很骨感：临床数据显示，药物产生的抗肌萎缩蛋白量微乎其微。在长达 48 周的步行测试中，安慰剂组和给药组的表现几乎没有拉开差距。监管机构的逻辑很简单：同情心不能代替科学证据。如果一款昂贵的罕见病药不能证明它确实延缓了患儿的残疾进程，那么它就不配留在市场上。四、 PTC 的至暗时刻：管线告急与战略焦虑 Translarna 的失败对 PTC 来说是毁灭性的打击。作为公司的核心支柱，这款药曾贡献了可观的现金流。连带打击：去年，PTC 另一款治疗弗里德里希共济失调的候选药 vatiquinone 也因有效性证据不足被 FDA 拒之门外。信心崩塌：投资者开始质疑 PTC 处理复杂临床数据的能力。在 DMD 领域，随着基因疗法（如 Elevidys）的入场，Translarna 这种效力模糊的口服药正迅速失去生存空间。未来的赌注： PTC 目前被迫转向更早期的管线，但研发资金的紧缺已成为其悬在头上的达摩克利斯之剑。结语：科学不需要“安慰剂” Translarna 的离场，标志着罕见病研发中“靠微弱数据博取同情分”的时代彻底结束。对于那些等待了十年的 DMD 家庭来说，这无疑是沉痛的一击。但从行业长远发展来看，FDA 和 EMA 的联手封杀，实际上是在清理赛道，将资源引向那些真正具有“实质性证据”的创新疗法。 2026 年，对于 PTC 来说是断臂求生的一年。而对于医疗行业来说，这再次证明了一个真理：在生与死的临床战场上，唯有硬核的数据，才是唯一的入场券。欢迎在评论区留下你的评论。高质量观点，我们会精选置顶。我们将持续输出：洞察行业变局，预判未来先机。我们持续深耕医疗健康赛道，为您提供：深度解读：穿透表象，还原商业底层逻辑。标的拆解：拆解并购核心，寻找估值锚点。趋势研判：捕捉投融资风向，预警行业巨震。把复杂问题，讲清楚。【互动环节】你认为 FDA 对这款已在欧洲销售 12 年的药物执行“零容忍”是保护了患者，还是剥夺了患者最后的尝试机会？在罕见病领域，我们该如何平衡“监管严谨性”与“患者迫切需求”？评论区留下你的深度思考！ 👇 访问网站：https://www.briinsight.com 了解更多👇 👇 商务合作与咨询，敬请扫描二维码 👇 生物医药前沿洞察｜职业成长加速器聚焦创新药、制剂技术、CDMO、AI药物研发等领域的最新突破；专注生物医药研发、注册申报、临床进展与市场趋势解析；汇聚业内声音、人才资讯与实战经验，打造最具价值的行业交流平台。 👇 更多精彩内容，敬请关注“Briinsight” 👇 一年仅需两针！吉利德 Yeztugo 开启 HIV 预防“降维打击”模式：药王归位，千亿市场面临洗牌！ Incyte 2025 战报：营收突破 50 亿！裁员缩线后，首席执行官为何对一款“面霜”寄予厚望？裁员 15% 后的“渤健式”反击：10 项 III 期临床、10 亿美金节省，阿兹海默新药暴涨 54%，老牌药王正在“换血”！ FDA的“20%红线”与中国的“加速度”：270亿美元临床市场洗牌，谁在抢夺美国吃不下的蛋糕？

100 项与爱塔乐伦相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D09323	爱塔乐伦	M09AX03	DB05016

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
杜氏肌营养不良症	欧盟	PTC Therapeutics International Ltd.	2014-07-31
杜氏肌营养不良症	冰岛	PTC Therapeutics International Ltd.	2014-07-31
杜氏肌营养不良症	列支敦士登	PTC Therapeutics International Ltd.	2014-07-31
杜氏肌营养不良症	挪威	PTC Therapeutics International Ltd.	2014-07-31

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
囊性纤维化	申请上市	欧盟	PTC Therapeutics, Inc.	-
贝克型肌营养不良	临床3期	澳大利亚	PTC Therapeutics, Inc.	2012-05-20
贝克型肌营养不良	临床3期	比利时	PTC Therapeutics, Inc.	2012-05-20
贝克型肌营养不良	临床3期	加拿大	PTC Therapeutics, Inc.	2012-05-20
贝克型肌营养不良	临床3期	法国	PTC Therapeutics, Inc.	2012-05-20
贝克型肌营养不良	临床3期	德国	PTC Therapeutics, Inc.	2012-05-20
贝克型肌营养不良	临床3期	以色列	PTC Therapeutics, Inc.	2012-05-20
贝克型肌营养不良	临床3期	意大利	PTC Therapeutics, Inc.	2012-05-20
贝克型肌营养不良	临床3期	西班牙	PTC Therapeutics, Inc.	2012-05-20
贝克型肌营养不良	临床3期	瑞典	PTC Therapeutics, Inc.	2012-05-20

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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


NCT03179631 \| NCT01826487 \| NCT00592553 (Study 041 \| Study 007 \| ACT DMD, AAN2024)	临床2/3期	杜氏肌营养不良症	-	Ataluren	範夢襯築網鹽齋壓衊艱(衊願鑰廠鬱鏇蓋齋選鬱) = 獵醖鑰觸鏇膚淵膚範淵製膚淵鑰壓鬱鹽鹽蓋鹽 (選遞艱齋構艱淵膚襯鑰 ) 更多	积极	2024-04-09
NCT04336826 (CTgov)	临床2期	杜氏肌营养不良症	6	Ataluren	構鬱鹹製窪膚憲膚膚窪 = 鏇醖餘鹹齋鏇獵鏇獵鑰鏇壓鏇築壓積鏇壓獵積 (範艱製膚願網鹹壓鬱淵, 夢餘廠齋遞壓獵鏇鑰繭 ~ 壓簾壓鬱廠襯窪獵鏇獵) 更多	-	2024-04-01
NCT03179631 (Study 041, AAN2023)	临床3期	杜氏肌营养不良症 nonsense mutation (nm) DMD	-	Ataluren	淵憲鏇膚糧鹽繭壓顧鹹(範網願壓膚蓋顧淵選憲) = 糧憲繭鹽網鏇遞繭膚積憲願窪壓獵糧遞窪願糧 (觸醖繭鏇糧遞醖簾壓願 ) 更多	积极	2023-04-25
NCT03179631 (Study 041, MDA2023)	临床3期	杜氏肌营养不良症	701	Ataluren	壓膚襯憲艱夢壓衊淵鹽(選憲獵糧鏇糧製衊繭網) = 淵範醖淵築憲膚遞繭窪積鬱膚築築構鹹壓繭簾 (鏇窪齋鏇廠淵齋積築願 ) 更多	积极	2023-03-19
NCT03179631 (Study 041, MDA2023)	临床3期	杜氏肌营养不良症	-	Ataluren	範獵衊鬱範蓋繭鏇構鑰(鏇齋鑰憲鹽築齋淵廠獵) = 簾鹹蓋艱淵願糧積廠廠窪壓觸壓糧願艱醖餘餘 (構範衊鑰廠遞鑰繭網鹽 ) 更多	积极	2023-03-19
NCT00803205 \| NCT02139306 \| NCT02107859 (Pubmed \| Cochrane Database Syst Rev)	N/A	囊性纤维化	517	Ataluren and similar compounds	獵齋鹽築遞糧憲繭選範(構製顧製遞繭醖鑰鏇艱) = Ataluren was associated with a higher rate of episodes of renal impairment (risk ratio 12.81, 95% confidence interval 2.46 to 66.65; P = 0.002; I2 = 0%; 2 trials, 517 participants) 艱鬱鹽夢齋構繭獵糧蓋 (淵糧醖蓋積蓋網餘鬱願 ) 更多	-	2023-03-03
	N/A	囊性纤维化	517	Placebo		-	2023-03-03
NCT02647359 (STAR, CTgov)	临床2期	无虹膜	39	Ataluren (Ataluren)	積襯選選憲夢廠製遞壓(鹽鹽糧糧鹹淵鬱製餘壓) = 憲衊鑰製艱鑰壓齋衊鹹簾廠築糧網積憲構願鏇 (糧鏇獵糧顧襯鹽鹽構鑰, 7.425) 更多	-	2022-05-27
NCT02647359 (STAR, CTgov)	临床2期	无虹膜	39	Placebo+Ataluren (Placebo)	積襯選選憲夢廠製遞壓(鹽鹽糧糧鹹淵鬱製餘壓) = 醖鬱積積願夢衊顧壓憲簾廠築糧網積憲構願鏇 (糧鏇獵糧顧襯鹽鹽構鑰, 11.809) 更多	-	2022-05-27
NCT03796637 (CTgov)	临床2期	杜氏肌营养不良症	6	Ataluren	選選醖齋餘築膚獵觸願(齋憲鹹範網夢鏇簾網觸) = 齋衊壓範齋鏇鬱蓋壓壓糧製鹽夢膚築齋顧願願 (構鹽積齋顧選範壓顧蓋, 0.05874) 更多	-	2022-04-05
NCT03648827 (CTgov)	临床2期	杜氏肌营养不良症	20	Ataluren	鹹襯齋齋膚餘鑰鬱襯構(襯蓋構醖願築襯憲齋範) = 醖積膚壓蓋願餘製齋顧糧廠鏇積觸艱齋餘夢製 (製鬱願糧顧糧選鏇衊選, 淵憲鏇繭衊積積襯蓋顧 ~ 鬱窪鑰鹽廠蓋餘襯範壓) 更多	-	2022-04-05
NCT01557400 (Study 019, MDA2021)	临床3期	杜氏肌营养不良症 nonsense mutation	94	Ataluren 10, 10, 20 mg/kg	膚簾窪衊蓋壓夢蓋襯齋(窪積願膚膚鏇遞淵構艱) = a trend in delay in decline to predicted FVC <50% by ~1 year 範壓製蓋衊壓壓餘蓋膚 (觸築構願製鏇齋夢製膚 )	积极	2021-03-01
NCT01557400 (Study 019, MDA2021)	临床3期	杜氏肌营养不良症 nonsense mutation	94	Ataluren		积极	2021-03-01