A Phase 2, Double-Blind Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Efficacy, and Safety of Apitegromab in Subjects <2 Years Old With Spinal Muscular Atrophy (SMA)

This double-blind, Phase 2, multiple-dose study will be conducted to evaluate the PK/PD, efficacy, safety, and tolerability of apitegromab in subjects <2 years old with 5q autosomal recessive SMA who have delayed motor milestones for their age attributed to SMA at the discretion of the Investigator or a Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND) score <55.

开始日期2025-09-15

申办/合作机构

Scholar Rock, Inc.

NCT05861999

/ Recruiting临床4期

A Phase IV Open-Label Study Evaluating the Effectiveness and Safety of Risdiplam Administered in Pediatric Patients With Spinal Muscular Atrophy Who Experienced a Plateau or Decline in Function After Gene Therapy

This is an open-label, single-arm, multicenter clinical study to evaluate the effectiveness and safety of risdiplam administered in pediatric participants with SMA and 2 SMN2 copies who previously received onasemnogene abeparvovec and experience a plateau or decline in function. Participants to be enrolled are children <2 years of age genetically diagnosed with SMA.

开始日期2024-08-14

申办/合作机构-

NCT05861986

/ Recruiting临床4期

A Phase IV Open-Label Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Patients With Spinal Muscular Atrophy After Gene Therapy

This is an open-label, single-arm, multicenter clinical study to evaluate the effectiveness and safety of risdiplam administered as an early intervention in pediatric participants with spinal muscular atrophy (SMA) and 2 SMN2 copies who have previously received onasemnogene abeparvovec. Participants are children < 2 years of age genetically diagnosed with SMA.

开始日期2024-05-30

申办/合作机构-

100 项与利司扑兰相关的临床结果

登录后查看更多信息

100 项与利司扑兰相关的转化医学

登录后查看更多信息

100 项与利司扑兰相关的专利（医药）

登录后查看更多信息

259

项与利司扑兰相关的文献（医药）

2026-04-01·NEUROMUSCULAR DISORDERS

Longitudinal assessment of respiratory status utilising the amended Great Ormond Street Respiratory Score in treated spinal muscular atrophy type 1 children

Article

作者: Patelis, Vasileios ; Muntoni, Francesco ; Stimpson, Georgia ; Edel, Lisa ; Baranello, Giovanni ; Chan, Elaine ; Scoto, Mariacristina

Spinal muscular atrophy type 1 phenotypes have changed since the introduction of disease modifying therapies. The Great Ormond Respiratory Score has been used to assess respiratory function in children with spinal muscular atrophy. This paper presents an updated version of the score and aims to describe preliminary data reviewing the two-year changes of respiratory status in a cohort of spinal muscular atrophy type 1 patients treated with monotherapy or sequential disease modifying treatments. Patients were assessed using the amended Great Ormond Respiratory score at baseline, 6 months, 12 months and 24 months. 59 patients were reviewed. In total, 32 first initiated nusinersen therapy, 16 first initiated onasemnogene abeparvovec and 11 first initiated risdiplam therapy. There was a significant difference in baseline Great Ormond Respiratory Score between the nusinersen group and the onasemnogene abeparvovec group (p = 0.002). Risdiplam data is limited and is identified as preliminary. The data identifies treatment prior to 6 weeks of age stabilise at a lower score, with this being impacted by age of treatment. Those treated after one year show worse scores but stabilise. Despite the switching between disease modifying treatments, the Great Ormond Respiratory Score is a useful and easy-to-use clinical outcome measure which can demonstrate change in respiratory status over longitudinal review.

2026-04-01·NEUROBIOLOGY OF DISEASE

Skeletal muscle in spinal muscular atrophy: Critical insights from pathogenesis to therapeutic strategies

Review

作者: Gagliardi, Delia ; Panicucci, Chiara ; Moggio, Maurizio ; Comi, Giacomo Pietro ; Ripolone, Michela ; Napoli, Laura ; Magni, Giulia ; Corti, Stefania Paola ; Bruno, Claudio ; Ottoboni, Linda

Spinal muscular atrophy (SMA) is a genetic neuromuscular disorder caused by loss of the survival motor neuron (SMN) protein. While SMA was originally viewed as a pure motor neuron disease, it is currently considered a multi-system disorder in which skeletal muscle plays a pathogenic role. Muscular defects, such as impaired myogenesis and mitochondrial dysfunction, contribute to pathogenesis partly independently of denervation. Accumulating evidence suggests that the SMN deficit impairs muscle development from the earliest stages of fetal life, with delayed myotube maturation and modification of the expression of myogenic regulatory factors. This leads to pathology characterized by selective fiber atrophy, metabolic disturbances, and severe involvement of axial and intercostal musculature with relative sparing of the diaphragm. Furthermore, despite the revolutionary therapeutic effects of nusinersen, risdiplam, and onasemnogene abeparvovec, skeletal muscle abnormalities remain frequent, particularly in symptomatic patients, highlighting the need for muscle-directed therapies. Of the current candidate approaches, myostatin inhibition, targeting a negative regulator of muscle mass, is the most clinically advanced, while other strategies such as mitochondrial protection remain at earlier developmental stages. Work with neuromuscular models and stem cell-derived organoids continues to shed light on the SMN-mediated interactions between muscle and nerve. Collectively, these findings indicate that skeletal muscle is both a key driver of SMA pathology and an essential target for novel therapies.

2026-03-01·NEUROLOGICAL SCIENCES

Patient reported outcome measures in spinal muscular atrophy and duchenne muscular dystrophy: review of instruments and their inclusion in clinical and regulatory processes

Review

作者: Spataro, Clarissa ; D'amico, Adele ; Scopinaro, Annalisa ; Sansone, Valeria ; Meregaglia, Michela ; Malandrini, Francesco ; Ciani, Oriana

Abstract:

Objectives:

This study aims to analyze the use of patient-reported outcomes measures (PROMs), observer-reported outcome measures (ObsROMs), and caregiver-reported outcome measures (CROMs) in spinal muscular atrophy (SMA) and Duchenne muscular dystrophy (DMD). The objectives are twofold: (1) to identify and characterize available instruments to be used in research and clinical practice, and (2) to assess their inclusion in drug development and regulatory assessment processes.

Methods:

A systematic search was conducted using PubMed, Google Scholar, Scopus, and the ePROVIDE database to identify PROMs, ObsROMs, and CROMs for SMA and DMD. The identified instruments were analyzed for validation, psychometric properties, Minimal Clinically Important Difference (MCID), and recall period. Additionally, clinical trial protocols, relative study publications, European Public Assessment Report (EPAR), and Italian Medicines Agency (AIFA) reports for innovativeness recognition on medicines for SMA and DMD (i.e., nusinersen, onasemnogene abeparvovec, risdiplam, and ataluren) were reviewed to evaluate the inclusion of these measures in drug development and regulatory assessment.

Results:

The initial search identified 50 questionnaires, including 40 PROMs, 5 ObsROMs, and 5 CROMs. Of these, 15 (30.0%) instruments were included in pivotal clinical trial protocols, with none designated as primary endpoints. Only 6 (12.0%) instruments were mentioned in EPARs, and MCID determination was reported for 6 (12.0%) of the instruments. Generic instruments like the PedsQL were frequently used but criticized for limited specificity.

Conclusions:

Despite the availability of PROMs, ObsROMs, and CROMs for SMA and DMD, their use in clinical trials and regulatory documents is limited and inconsistent. Greater standardization and systematic inclusion of these measures are needed to support patient-centered drug development and evaluation.

598

项与利司扑兰相关的新闻（医药）

2026-02-23

·学平E药投研

为什么中外制药成为日本市值第一的药企？

2026年初，一家并非日本历史最悠久、规模也并非最大的制药企业——中外制药（Chugai Pharmaceutical），以超过1000亿美元的市值，稳居日本制药行业第一宝座。这一成绩不仅超越了拥有240余年历史的武田制药，也将近年来风头正劲的第一三共甩在身后。更令人瞩目的是，中外制药的崛起并非依靠传统的规模扩张或并购整合，而是通过一种独特的"战略联盟+自主创新"模式实现的。这一路径为全球制药行业，尤其是中国创新药企，提供了极具价值的战略参考。第一章：历史转折——2002年罗氏联盟的深远影响1.1 危机中的战略抉择中外制药成立于1925年，虽历史悠久，但在20世纪末已陷入发展瓶颈。面对全球制药行业集中度提升和创新成本飙升的压力，2002年，中外制药做出了一个改变命运的决策：接受瑞士罗氏制药16亿美元投资，出让50.1%股权，成为罗氏控股子公司。然而，这并非一次简单的"卖身"。双方约定了一种非传统的战略合作关系：• 管理独立性保留：中外制药在管线研发上保持独立决策权，避免了大公司并购后的创新僵化• 资源深度整合：罗氏日本分公司与中外制药合并，研发团队共享数据和全球研发进度• 利益长期绑定：罗氏通过股权增值获益，而非短期财务回报，双方形成真正的命运共同体1.2 联盟模式的复利效应这一联盟在20余年间产生了惊人的复利效应。2002年罗氏入股时，中外制药市值约30亿美元；2026年稳居日本第一时已达1000亿美元。罗氏的初始投资如今价值已增长超过30倍，而中外制药也借助罗氏的全球化网络，实现了从本土药企到全球创新者的蜕变。第二章：创新引擎——技术平台与管线布局2.1 抗体技术的深耕中外制药的成功，核心在于其在抗体工程领域建立的难以复制的技术护城河。与许多跟风热门靶点的药企不同，中外制药专注于复杂生物制剂的开发。其代表性产品包括：血友病领域的Hemlibra（艾美赛珠单抗），采用双特异性抗体技术，已成为全球 blockbuster 并改变了治疗标准；肿瘤领域的Alecensa和Tecentriq，使中外制药成为日本肿瘤市场领导者；罕见病领域的Evrysdi（脊髓性肌萎缩症口服药），以便利性颠覆市场；眼科领域的Vabysmo，通过双抗机制延长给药间隔；以及减肥代谢领域的Orforglipron，作为口服小分子GLP-1受体激动剂，III期临床数据积极，被视为未来增长引擎。2.2 差异化创新策略中外制药的管线策略体现了清晰的差异化思维。当全球药企扎堆PD-1、GLP-1多肽药物时，中外制药选择开发口服小分子GLP-1药物Orforglipron，以及双抗、ADC等复杂生物制剂，形成技术壁垒。同时针对日本老龄化社会特点，深耕肿瘤、眼科、骨质疏松等慢病领域，并通过罕见病药物建立高定价能力。借助罗氏的全球临床网络，实现产品日本与欧美同步上市，最大化市场价值。第三章：财务表现——盈利质量驱动估值3.1 业绩的持续增长中外制药的财务数据验证了其商业模式的可持续性。2020年至2024年间，营业收入从7,792亿日元增长至11,663亿日元，年均增长率10.6%；净利润从2,078亿日元增长至3,865亿日元，年均增长率16.8%；净利润率从26.7%提升至33.1%；海外收入占比从约35%提升至46%。3.2 估值逻辑的转变资本市场给予中外制药高估值的核心逻辑在于四个方面：一是收入多元化，近半数收入来自海外，降低日本医保控费风险；二是高盈利能力，33%的净利润率在全球制药行业处于顶尖水平；三是可持续增长，管线储备丰富，Orforglipron等重磅产品即将上市；四是低风险模式，与罗氏的联盟提供了稳定的全球商业化保障。结语：新范式的胜利中外制药的崛起，标志着全球制药行业竞争逻辑的转变：规模不再是决定性因素，联盟的质量、创新的深度和财务的纪律性才是市值的核心驱动力。在创新药研发成本飙升、全球化门槛日益提高的今天，中外制药证明了一种中间道路的可行性——既非完全独立发展，也非被动被并购，而是通过精心设计的战略联盟，在保持创新活力的同时，借力全球资源实现价值最大化。对于中国药企而言，中外制药的经验表明：成为MNC的研发工厂或制造工厂是一条快速的崛起路径。

2026-02-21

·百度百家

2026年中国儿科用药行业市场规模及投资前景预测分析报告

第一章、中国儿科用药行业市场概况中国儿科用药行业近年来呈现出稳健增长态势，其发展既受到人口结构变化、医疗政策优化及儿童健康需求升级的多重驱动，也面临药物研发难度大、剂型适配性不足、临床试验开展受限等结构性挑战。截至2025年，全国0–14岁儿童人口规模为2.29亿人，占总人口比重为16.3%，较2024年的2.31亿人小幅下降0.86%，但儿童人均门诊就诊次数达3.8次/年，较2024年的3.6次上升5.6%，反映出基层儿科医疗服务可及性提升与家长健康意识增强的双重效应。在诊疗端，全国登记在册的儿童专科医院数量达176家，综合医院中设立独立儿科门诊的比例为92.4%，较2024年的90.7%提高1.7个百分点；儿科执业医师总数为16.8万人，每千名儿童拥有儿科医师0.73名，虽较2024年的0.71名有所改善，但仍低于国家卫生健康委员会设定的2025年“每千儿童配备0.85名儿科医师”的阶段性目标。从市场规模看，2025年中国儿科用药整体市场规模达1,248.6亿元，同比增长9.3%，增速高于全医药市场整体7.1%的增幅。化学药占比58.2%，达726.5亿元；中成药占比32.7%，达408.3亿元；生物制剂及其他新兴治疗手段（如单抗类、基因治疗辅助用药）占比9.1%，达113.8亿元，成为增长最快的细分板块，同比增幅达24.6%。按治疗领域划分，呼吸系统用药仍居首位，2025年规模为392.4亿元，占整体31.4%；其次为消化系统用药（186.3亿元，14.9%）、抗感染用药（178.5亿元，14.3%）、神经系统用药（102.7亿元，8.2%）及营养补充与免疫调节类用药（95.1亿元，7.6%）。值得注意的是，儿童罕见病用药市场在2025年实现突破性进展，获批上市的儿童专用罕见病治疗药物达14个，覆盖脊髓性肌萎缩症（SMA）、黏多糖贮积症Ⅱ型、苯丙酮尿症等8类疾病，对应市场规模为33.6亿元，同比增长41.2%，显著高于行业均值。产品结构方面，2025年国内儿科用药中口服液体制剂占比最高，达42.5%，片剂（含分散片、咀嚼片）占23.1%，颗粒剂占15.8%，注射剂占12.3%，外用及其他剂型合计占6.3%。剂型适配性持续改善，已获批的儿童专用规格药品数量达2,147个，较2024年的1,983个增长8.3%；其中通过国家儿童用药优先审评审批通道获批的产品达137个，占全年新增儿童适用药品总数的64.6%。政策层面，2025年《儿童基本药物目录（2025年版）》正式实施，共收录儿童适宜品种586种，较2022年版增加124种，覆盖率达89.3%；医保谈判向儿童用药倾斜力度加大，2025年新增纳入国家医保目录的儿童专用药品达29个，平均降价幅度为52.7%，其中利司扑兰口服溶液用散、诺西那生钠注射液等高值罕见病用药首次实现医保全额报销。从企业格局看，2025年市场集中度进一步提升，前十大企业合计市场份额达46.8%，较2024年的44.2%上升2.6个百分点。华润三九以87.3亿元儿科用药销售收入位居同比增长11.5%；第二位为扬子江药业集团，达72.6亿元，同比增长9.8%；第三位为齐鲁制药，达58.4亿元，同比增长13.2%；其他主要参与者包括恒瑞医药（45.9亿元）、科伦药业（39.7亿元）、辰欣药业（33.2亿元）、一品红药业（31.5亿元）、健民集团（28.6亿元）、葫芦娃药业（26.4亿元）和正大天晴（24.8亿元）。值得注意的是，葫芦娃药业作为专注儿童用药的代表性企业，其2025年儿童专用剂型产品收入占比达98.7%，核心产品小儿肺热咳喘口服液、维D2磷葡钙片等在公立医疗机构终端占有率分别达22.4%和18.9%，稳居细分品类第一。根据博研咨询&市场调研在线网分析，供应链与流通环节亦呈现专业化趋势：2025年全国具备儿童用药GMP认证资质的生产企业共412家，较2024年净增23家；儿童用药专用物流温控运输覆盖率已达86.5%，其中冷链运输达标率由2024年的79.3%提升至83.7%；在零售终端，连锁药店中设立“儿童健康专区”的门店比例达68.2%，较2024年提升9.4个百分点，TOP20连锁药房儿童用药SKU平均达187个，同比增长12.6%。中国儿科用药行业正处于从“供给短缺”向“结构优化+质量跃升”转型的关键阶段，2026年预计市场规模将达1,365.2亿元，同比增长9.4%，在创新研发加速、医保支付扩容、分级诊疗深化及家庭健康支出意愿持续增强的共同作用下，行业正逐步构建起覆盖预防、诊断、治疗、康复全周期的儿童用药生态体系。第二章、中国儿科用药产业利好政策中国儿科用药产业近年来持续获得国家层面系统性政策支持，已形成涵盖研发激励、审评加速、医保准入、临床推广及生产保障在内的全链条扶持体系。2025年，国家药监局共受理儿童用药注册申请417件，其中新药临床试验（IND）申请186件，新药上市许可（NDA）申请93件，仿制药一致性评价补充申请138件，较2024年的352件增长18.5%；在审评环节，儿童用药优先审评通道平均审评时限为112个工作日，较常规通道缩短58.3%，全年通过该通道获批的儿童专用新药达37个，占全部获批儿童新药总数的72.5%。自2021年《鼓励儿童用药加快上市审评工作举措》实施以来，截至2025年底，累计已有156个儿童适宜剂型或新增儿童适应症获批，覆盖抗肿瘤、内分泌、神经精神、遗传代谢等12类重大儿童疾病领域。在医保支付端，2025年国家医保药品目录调整中，儿童用药专项评审机制全面落地，设置独立评审组与临床价值权重系数（儿童适应症权重为1.3，高于成人通用系数1.0）。全年新增纳入目录的儿童专用药品达29个，涉及17个治疗类别，平均降价幅度为52.7%，其中12个为国产首研儿童新药，如利司扑兰口服溶液用散降价56.4%、布洛芬混悬液（30mg/5mL）降价48.2%。2025年儿童用药在医保基金支出中的占比为6.8%，较2024年的6.1%提升0.7个百分点；全国公立医疗机构儿童用药医保实际报销比例加权平均达73.4%，较2024年的70.2%提高3.2个百分点，其中中西部地区提升尤为显著，甘肃、贵州、云南三省儿童用药平均报销比例分别达76.8%、75.3%和74.9%。 1. 国家级专项财政投入与研发资助 2025年，科技部“十四五”国家重点研发计划“常见多发病防治研究”重点专项中，专设儿童用药攻关方向，立项支持项目23项，总经费达4.28亿元，较2024年的3.65亿元增长17.3%；其中单个项目最高资助额度为3,800万元，用于支持儿童罕见病靶向药物临床前研究与早期临床试验。国家卫健委联合工信部设立的“儿童用药关键技术攻关与产业化项目”，2025年下达专项资金2.96亿元，支持14家单位开展儿童剂量学建模、掩味技术、微球缓释制剂、纳米晶分散片等核心工艺攻关，已建成儿童用药专用技术平台7个，完成中试转化品种31个，其中19个于2025年内实现产业化落地。 2. 儿童基本药物目录与临床应用规范建设《儿童基本药物目录（2025年版）》于2025年3月1日正式施行，共收录586个品种、823个品规，较2022年版新增124个品种、187个品规，儿童适宜剂型覆盖率由76.4%提升至89.3%。目录中明确标注“限儿童使用”的品种达217个，占比37.0%；标注“儿童用法用量”的品种达532个，占比90.8%。同步发布的《儿童用药临床应用指导原则（2025年修订版）》首次将体重-体表面积双参数给药模型写入强制性推荐条款，要求二级及以上医院儿科门诊100%执行该模型，基层医疗卫生机构执行率达86.7%，较2024年的72.1%大幅提升。2025年全国三级医院儿童用药合理率（依据WHO合理用药指标评估）达89.4%，较2024年的86.2%提高3.2个百分点；基层医疗机构该指标为78.3%，同比提升4.5个百分点。 3. 生产供应保障与质量监管强化 2025年，工信部牵头建立“儿童用药保供稳价监测平台”，覆盖全国412家儿童用药生产企业、3,267家流通企业及12.8万家终端医疗机构，实现从原料采购、产能负荷、库存水位到终端缺货预警的全链路动态监控。全年触发黄色预警（库存低于30天）事件142起，红色预警（库存低于7天）事件19起，均在72小时内完成跨区域调拨响应，缺货平均恢复时长为38.6小时，较2024年的52.3小时缩短26.2%。在质量监管方面，国家药监局对儿童用药开展专项抽检，2025年共抽检样品4,826批次，合格率为99.62%，较2024年的99.47%提升0.15个百分点；其中口服液体剂型合格率99.75%，颗粒剂99.68%，注射剂99.51%，均高于全品类药品抽检平均合格率（99.34%）。 4. 医疗服务协同与基层能力提升 2025年，国家卫生健康委员会持续推进“县域儿科振兴计划”，在全国遴选216个县开展儿科能力标准化建设，累计投入中央财政补助资金13.7亿元，为县级医院新建或改扩建标准化儿童诊疗中心328个，配备儿童专用检验设备（如微量血气分析仪、新生儿黄疸检测仪）4,127台，培训基层儿科医师38,642人次，人均培训时长126学时。截至2025年底，全国85.3%的县域医共体已实现儿童常见病诊疗指南统一、处方流转互通、检查结果互认；基层医疗机构儿童呼吸道感染规范诊疗率由2024年的64.8%提升至77.2%，腹泻病补液治疗规范率由68.5%提升至82.9%。 5. 创新生态与国际合作支持 2025年，国家药监局与美国FDA、欧盟EMA签署《儿童用药国际协调合作备忘录》，推动中国儿童临床试验数据国际互认。全年有21个国产儿童新药启动国际多中心临床试验，涉及12个国家，其中8个进入III期阶段；恒瑞医药的HR-19034（儿童难治性癫痫新分子实体）获FDA授予“儿科罕见病资格认定（PRV）”，成为国内第5个获此资格的儿童用药。同期，海南博鳌乐城国际医疗旅游先行区批准进口未在国内上市的儿童专用创新药14个，包括诺华的沙库巴曲缬沙坦钠儿童颗粒剂、罗氏的利妥昔单抗儿童预充式注射液等，2025年乐城儿童用药临床真实世界数据（RWD）采集量达12,847例，支撑3个品种完成国内上市申报。第三章、中国儿科用药行业市场规模分析中国儿科用药行业市场规模已进入结构性扩张与高质量增长并行的新阶段，其增长动能正由人口基数驱动转向临床需求深化、支付能力提升、产品结构升级与政策红利释放的多重叠加。2025年，全国儿科用药市场规模达1,248.6亿元，较2024年的1,142.3亿元增长9.3%，高于同期全医药工业整体7.1%的增速，也显著高于化学药板块6.4%和中成药板块5.9%的增长水平。从复合增速看，2021–2025年五年间行业CAGR为8.7%，其中2023年受集采扩围及部分老品种降价影响增速阶段性放缓至6.2%，而2024–2025年在创新药放量、医保新增准入及基层诊疗下沉带动下重回双位数增长轨道。 1. 按产品类型划分的市场规模与结构演变 2025年，化学药仍为最大细分板块，规模为726.5亿元，占整体58.2%，同比增长8.9%；中成药规模为408.3亿元，占比32.7%，同比增长8.5%；生物制剂及其他新兴治疗手段（含单抗、融合蛋白、基因治疗辅助用药等）规模为113.8亿元，占比9.1%，同比增长24.6%，增速连续三年保持行业第一。值得注意的是，儿童专用生物类似药于2025年实现零的突破，首个获批的贝伐珠单抗儿童适应症注射液（用于儿童视网膜母细胞瘤辅助治疗）当年即实现销售收入3.2亿元，带动生物制剂板块中“儿童专属新分子实体”类目占比由2024年的31.4%提升至38.7%。按剂型统计，口服液体制剂（含糖浆、混悬液）2025年规模为530.7亿元，占比42.5%，同比增长10.1%；颗粒剂221.9亿元（17.8%），同比增长7.3%；分散片与咀嚼片合计162.4亿元（13.0%），同比增长11.8%，成为增速最快的固体口服剂型；注射剂153.3亿元（12.3%），同比增长5.2%，增速放缓主因静脉输注管理趋严及门诊手术比例上升导致住院用药减少。 2. 按治疗领域划分的市场格局与增长极分布呼吸系统用药继续领跑，2025年规模为392.4亿元，占整体31.4%，同比增长8.6%，其中小儿肺热咳喘口服液、金振口服液、孟鲁司特钠颗粒三大单品合计市占率达29.7%；消化系统用药186.3亿元（14.9%），同比增长7.9%，以酪酸梭菌二联活菌散、消旋卡多曲颗粒为代表的产品在基层腹泻病防治中渗透率持续提升；抗感染用药178.5亿元（14.3%），同比增长6.4%，但受国家抗菌药物临床应用专项整治影响，头孢克肟颗粒、阿奇霉素干混悬剂等传统大品种增速明显放缓，而新型β-内酰胺酶抑制剂复方制剂（如哌拉西林他唑巴坦儿童剂型）实现23.5%高增长；神经系统用药102.7亿元（8.2%），同比增长12.4%，主要受益于儿童ADHD诊疗规范化推进及盐酸托莫西汀口服溶液、哌甲酯缓释片等医保放量；营养补充与免疫调节类用药95.1亿元（7.6%），同比增长15.3%，维生素D衍生物、益生菌复合制剂、人血丙种球蛋白儿童规格等品类呈现消费升级特征。 3. 按终端渠道划分的销售分布与增长差异 2025年，公立医院仍是最大终端，销售额为782.4亿元，占比62.7%，同比增长7.8%，但占比连续第五年下降（2021年为71.3%）；零售药店渠道达328.6亿元，占比26.3%，同比增长13.2%，其中连锁药店贡献274.1亿元（占零售总额83.4%），单店儿童用药平均SKU达187个，同比增长12.6%；线上渠道（含B2C平台、O2O即时配送、互联网医院处方外流）达137.6亿元，占比11.0%，同比增长28.4%，增速连续三年超25%，其中京东健康、阿里健康、平安好医生三大平台儿童用药GMV分别为42.3亿元、38.7亿元和29.5亿元，合计占线上总规模的80.5%。值得关注的是，“互联网+儿科专科服务”模式加速成熟，2025年接入国家医保信息平台的互联网医院中，具备儿童在线问诊与电子处方能力的达1,247家，全年开具儿童电子处方3,826万张，其中42.6%完成线下取药或O2O配送，直接带动相关药品销售增长19.7%。 4. 区域市场分化与下沉潜力 2025年，华东地区仍为最大区域市场，规模为412.3亿元，占全国33.0%，同比增长8.9%；华南地区286.5亿元（22.9%），同比增长10.2%；华北地区217.8亿元（17.4%），同比增长7.5%；中西部地区合计331.0亿元（26.5%），同比增长11.8%，增速首次全面超越东部，其中河南、四川、湖北三省儿童用药市场规模分别达68.4亿元、62.9亿元和57.3亿元，位列全国前五。县域及乡镇市场表现尤为突出：2025年县级医院儿童用药销售额达246.7亿元，同比增长13.5%；乡镇卫生院及社区中心达112.8亿元，同比增长16.2%，增速为城市三级医院（7.1%）的两倍以上，反映分级诊疗制度与“县域儿科振兴计划”成效显著。 5. 未来六年市场规模预测与增长动力拆解基于人口结构趋势、疾病谱变化、医保扩容节奏、创新药上市周期及基层服务能力提升速率等多维变量建模，预计2026–2032年中国儿科用药市场规模将保持稳健上行态势。2026年市场规模预计达1,365.2亿元，同比增长9.4%；2027年为1,490.1亿元，同比增长9.1%；2028年为1,624.3亿元，同比增长9.0%；2029年为1,768.5亿元，同比增长8.9%；2030年为1,923.6亿元，同比增长8.8%；2031年为2,090.2亿元，同比增长8.7%；2032年为2,269.8亿元，同比增长8.6%。七年复合增长率（CAGR）为9.0%，略高于2021–2025年8.7%的历史均值，表明行业已进入可持续增长通道。增长动力中，创新药贡献率将由2025年的28.3%提升至2032年的41.6%，医保新增准入贡献率稳定在22–25%，基层诊疗量提升贡献率由2025年的19.4%升至2032年的26.1%，而传统大品种自然增长贡献率则由32.3%逐步降至10.7%。第四章、中国儿科用药市场特点与竞争格局分析中国儿科用药市场已形成“高政策敏感性、强临床依附性、低剂型适配率、中度集中化”的典型结构性特征，其竞争格局正经历从“渠道驱动”向“临床价值+技术壁垒+生态协同”三维竞争范式的深刻转型。2025年，行业CR10（前十大企业市场份额）达46.8%，较2024年的44.2%提升2.6个百分点，但CR3（前三强）为21.7%，较2024年的21.1%仅微增0.6个百分点，表明头部企业虽持续扩大规模优势，但第二梯队企业加速追赶，市场竞争烈度不降反升。尤为关键的是，2025年儿童专用剂型产品在整体儿科用药销售中的占比为68.3%，较2024年的65.1%提升3.2个百分点，但距离《“十四五”医药工业发展规划》提出的“2025年儿童适宜剂型覆盖率超85%”目标仍有差距，凸显剂型创新仍是行业核心瓶颈。 1. 市场结构性特点深度解析儿科用药具有显著的“小批量、多批次、高定制化”生产特征。2025年，全国儿童用药生产企业平均单品种年产量为2,847万支/瓶，仅为成人化学药均值（1.26亿支/瓶）的22.6%；而平均批次数达142批次/年，是成人药品（58批次/年）的2.4倍。这种生产模式直接推高单位成本——2025年儿童口服液体制剂平均制造成本为1.83元/支，较同成分成人规格片剂（0.47元/片）高出289.4%。在流通环节，儿童用药SKU复杂度更高：2025年全国公立医疗机构平均配备儿童药品种数为427个，但其中仅291个为高频使用品种（年使用量＞1万盒），其余136个为低频、急救或罕见病专用品种，库存周转天数达128天，显著高于成人药品的83天。临床端则呈现高度依赖指南与专家共识的特点：2025年三级医院儿科处方中，依据《国家儿科诊疗规范（2025年版）》开具的比例为89.4%，二级医院为78.3%，基层仅为62.1%，反映出临床行为标准化程度与机构层级强相关。 2. 企业竞争梯队与战略分化 2025年，中国儿科用药市场已清晰形成四大竞争梯队。第一梯队为综合型医药巨头，以华润三九、扬子江药业集团、齐鲁制药为代表，2025年合计销售收入达218.3亿元，占CR10总额的46.7%，其核心优势在于全品类覆盖能力与强大的终端准入能力。其中华润三九以87.3亿元居首，其儿童感冒、消化、营养三大类产品线收入占比达82.4%，小儿消积止咳口服液单品年销售额达14.2亿元，市场占有率为18.7%；扬子江药业集团72.6亿元中，63.5%来自呼吸与消化系统用药，依托其在全国3,267家县级医院的深度覆盖网络实现下沉放量；齐鲁制药58.4亿元收入中，有31.2亿元来自抗感染与神经系统用药，其头孢克肟颗粒、盐酸托莫西汀口服溶液在医保谈判后于2025年实现在全国92.4%的三级医院进院。第二梯队为专科型儿童药企，以葫芦娃药业、一品红药业、健民集团为核心，2025年合计收入111.5亿元，占CR10的23.9%。葫芦娃药业以26.4亿元位居该梯队首位，儿童专用剂型收入占比高达98.7%，其自主研发的掩味技术平台支撑17个儿童口感改良品种上市，小儿肺热咳喘口服液市占率达22.4%，维D2磷葡钙片达18.9%；一品红药业31.5亿元中，22.6亿元来自儿童呼吸与免疫调节领域，依托其与广州医科大学附属妇女儿童医疗中心共建的“儿童药物临床评价中心”，推动12个品种完成儿童适应症拓展；健民集团28.6亿元中，传统中成药占比71.3%，但其儿童专用小儿宣肺止咳颗粒、龙牡壮骨颗粒在2025年通过FDA GRAS认证，实现出口美国、加拿大等12国，海外收入达3.8亿元，同比增长47.2%。第三梯队为快速崛起的生物技术企业，以恒瑞医药、科伦药业、百奥泰为代表，2025年合计收入102.6亿元，占CR10的22.0%。恒瑞医药45.9亿元中，32.7亿元来自神经系统与肿瘤辅助用药，其自主研发的HR-19034（儿童难治性癫痫新分子实体）于2025年获FDA儿科罕见病资格认定，并在国内启动III期临床；科伦药业39.7亿元中，28.4亿元来自抗感染与营养支持类，其儿童用复方氨基酸注射液（18种）在2025年进入全国87.6%的儿童专科医院；百奥泰27.0亿元全部来自生物类似药，其贝伐珠单抗儿童适应症注射液2025年获批后迅速放量至3.2亿元，成为首个国产儿童肿瘤靶向用药。第四梯队为区域性龙头与新兴品牌，包括辰欣药业（33.2亿元）、正大天晴（24.8亿元）、华润双鹤（22.1亿元）、上海凯宝（19.7亿元）及近年快速成长的博瑞制药（17.3亿元）等，2025年合计收入137.1亿元，占CR10的29.4%，其策略聚焦于细分治疗领域突破与差异化渠道建设。辰欣药业在消化系统用药领域市占率达14.2%，其酪酸梭菌二联活菌散2025年销量达2.1亿袋；正大天晴在呼吸系统用药中以孟鲁司特钠颗粒为核心，覆盖终端达11.8万家药店；博瑞制药凭借儿童纳米晶技术平台，其布洛芬纳米混悬液2025年在华东地区三级医院占有率已达16.5%，并同步启动欧盟EMA注册。 3. 品牌认知与消费者行为变迁 2025年，家长对儿童用药的品牌信任度显著提升，全国城市家庭儿童用药首选品牌集中度（CR5）达53.7%，较2024年的50.2%提高3.5个百分点。在具体品类中，口服液体制剂首选品牌集中度最高，达61.4%，其中华润三九、葫芦娃、一品红三家合计占47.2%；颗粒剂为58.3%，健民集团、辰欣药业、正大天晴位列前三；而外用贴剂、栓剂等低频品类集中度较低，CR5仅为32.6%。消费者决策路径亦发生结构性变化：2025年，68.3%的家长在购药前会主动查询药品是否标注“儿童专用”及“明确年龄/体重用量”，较2024年的61.7%提升6.6个百分点；42.6%的家长愿意为口感改良（如无苦味、水果香型）支付15–30%溢价；线上购药用户中，83.4%会优先选择带有“儿科医生在线审方”标识的平台，京东健康、阿里健康、平安好医生三家平台2025年儿童用药复购率达52.7%，高于全品类平均复购率（38.9%）。 4. 区域竞争强度与渠道渗透差异

2026-02-19

·PRNewswire

PTC Therapeutics Provides Corporate Update and Reports Fourth Quarter and Full Year 2025 Financial Results

– Full-year 2025 product and royalty revenue of $831M, exceeding guidance – – Strong Sephience™ (sepiapterin) uptake since 2H 2025 launch with fourth quarter and 2025 revenue of $92M and $111M, respectively – – Cash of $1.95B as of December 31, 2025 – WARREN, N.J., Feb. 19, 2026 /PRNewswire/ -- PTC Therapeutics, Inc., (NASDAQ: PTCT) today announced a corporate update and financial results for the fourth quarter and full year ending December 31, 2025. "We delivered another strong quarter and finish to 2025, building on the successful global launch of Sephience," said Matthew B. Klein, M.D., Chief Executive Officer of PTC Therapeutics. "With our robust commercial engine, innovative R&D programs, and strong financial position, we look forward to continued success as we approach cash flow breakeven." Key Corporate Highlights Full-year 2025 product and royalty revenue of $831 million, exceeding guidance Global launch of Sephience off to strong start Q4 2025 revenue of $92 million, including $81 million revenue in the US and $11 million revenue ex-US Sephience total net revenue of $111 million in 2025 since launch 946 total patients on commercial therapy worldwide as of December 31, 2025 1,134 patient start forms received in the US as of December 31, 2025 Approval in Japan in December 2025; approval in Brazil in February 2026 Sephience global footprint expected to increase to 20 to 30 countries by end of 2026 In December 2025, PTC sold the remainder of its Evrysdi® (risdiplam) royalty to Royalty Pharma for $240 million upfront and up to $60 million in sales-based milestones; PTC maintains the right to receive a $150 million milestone from Roche based on single-year Evrysdi sales of $2.5 billion End-of-Phase 2 meeting with FDA held in Q4 2025 to discuss the votoplam Huntington's disease (HD) program Alignment reached on design of global Phase 3 trial, INVEST-HD, which is planned to initiate in 1H 2026 FDA confirmed openness for potential Accelerated Approval pathway given significant unmet need Type C meeting with FDA held in December 2025 to discuss the vatiquinone Friedreich's ataxia program; FDA indicated that an additional study would be necessary to support NDA resubmission and meeting minutes stated that this could be an open-label study with a natural history control group Fourth Quarter and Full Year 2025 Financial Highlights Total revenues were $164.7 million for the fourth quarter of 2025, compared to $213.2 million for the fourth quarter of 2024. Total revenues were $1,730.7 million for full year 2025, compared to $806.8 million for full year 2024. Included in total revenues is collaboration and license revenue of $998.4 million for the full year 2025, related to the votoplam license and collaboration agreement with Novartis, which closed in January 2025. Total net product revenues were $184.0 million for the fourth quarter of 2025, compared to $150.1 million for the fourth quarter of 2024. Total net product revenues were $586.7 million for full year 2025, compared to $582.1 million for full year 2024. Translarna™ (ataluren) net product revenues were $39.0 million for the fourth quarter of 2025, compared to $89.1 million for the fourth quarter of 2024. Translarna net product revenues were $235.3 million for full year 2025, compared to $321.1 million for full year 2024. Emflaza® (deflazacort) net product revenues were $27.1 million for the fourth quarter of 2025, compared to $50.5 million for the fourth quarter of 2024. Emflaza net product revenues were $146.4 million for full year 2025, compared to $207.2 million for full year 2024. Roche reported Evrysdi full year 2025 sales of approximately 1,757 CHF million, resulting in royalty revenue of $244.2 million to PTC for full year 2025, compared to $203.9 million to PTC for full year 2024. Based on U.S. GAAP (Generally Accepted Accounting Principles), GAAP R&D expenses were $133.1 million for the fourth quarter of 2025, compared to $124.8 million for the fourth quarter of 2024. GAAP R&D expenses were $455.2 million for full year 2025, compared to $534.5 million for full year 2024. Non-GAAP R&D expenses were $124.3 million for the fourth quarter of 2025, excluding $8.8 million in non-cash, stock-based compensation expense, compared to $116.0 million for the fourth quarter of 2024, excluding $8.8 million in non-cash, stock-based compensation expense. Non-GAAP R&D expenses were $419.6 million for full year 2025, excluding $35.7 million in non-cash, stock-based compensation expense, compared to $497.9 million for full year 2024, excluding $36.6 million in non-cash, stock-based compensation expense. GAAP SG&A expenses were $96.9 million for the fourth quarter of 2025, compared to $84.7 million for the fourth quarter of 2024. GAAP SG&A expenses were $347.1 million for full year 2025, compared to $300.9 million for full year 2024. Non-GAAP SG&A expenses were $87.2 million for the fourth quarter of 2025, excluding $9.7 million in non-cash, stock-based compensation expense, compared to $76.3 million for the fourth quarter of 2024, excluding $8.4 million in non-cash, stock-based compensation expense. Non-GAAP SG&A expenses were $308.3 million for full year 2025, excluding $38.9 million in non-cash, stock-based compensation expense, compared to $262.9 million for full year 2024, excluding $38.0 million in non-cash, stock-based compensation expense. Net loss was $135.0 million for the fourth quarter of 2025, compared to net loss of $65.9 million for the fourth quarter of 2024. Net income was $682.6 million for full year 2025, compared to net loss of $363.3 million for full year 2024. Cash, cash equivalents, and marketable securities were $1,945.4 million on December 31, 2025, compared to $1,139.7 million on December 31, 2024. Shares issued and outstanding as of December 31, 2025, were 81,474,366. Full Year 2026 Financial Guidance Total product revenue of $700 to $800 million, representing a 19 to 36% increase from 2025, with the majority from Sephience GAAP R&D and SG&A expense of $775 to $815 million Non-GAAP R&D and SG&A expense of $680 to $720 million, excluding estimated non-cash, stock-based compensation expense of $95 million Non-GAAP Financial Measures In this press release, the financial results of PTC are provided in accordance with GAAP and using certain non-GAAP financial measures. In particular, the non-GAAP R&D and SG&A expense financial measures exclude non-cash, stock-based compensation expense. These non-GAAP financial measures are provided as a complement to financial measures reported in accordance with GAAP because management uses these non-GAAP financial measures when assessing and identifying operational trends. In management's opinion, these non-GAAP financial measures are useful to investors and other users of PTC's financial statements by providing greater transparency into the historical and projected operating performance of PTC and the company's future outlook. Non-GAAP financial measures are not an alternative for financial measures prepared in accordance with GAAP. Quantitative reconciliations of the non-GAAP financial measures to their respective closest equivalent GAAP financial measures are included in the table below. Acronyms CHF: Confoederatio Helvetica Francs (Swiss francs) DMD: Duchenne muscular dystrophy FA: Friedreich's ataxia FDA: U.S. Food and Drug Administration GAAP: Generally Accepted Accounting Principles HD: Huntington's disease NDA: New Drug Application nmDMD: Nonsense mutation Duchenne muscular dystrophy PKU: Phenylketonuria R&D: Research and Development SG&A: Selling, General, and Administrative Today's Conference Call and Webcast Reminder To access the call by phone, please click here to register and you will be provided with dial-in details. The webcast conference call can be accessed on the Investors section of the PTC website at . A replay of the call will be available after completion of the call and will be archived on the company's website. About PTC Therapeutics, Inc. PTC is a global biopharmaceutical company dedicated to the discovery, development and commercialization of clinically differentiated medicines for children and adults living with rare disorders. PTC is advancing a robust and diversified pipeline of transformative medicines as part of its mission to provide access to best-in-class treatments for patients with unmet medical needs. The company's strategy is to leverage its scientific expertise and global commercial infrastructure to optimize value for patients and other stakeholders. To learn more about PTC, please visit and follow on LinkedIn, X, Instagram and Facebook. For more Information Investors Ellen Cavaleri +1 (615) 618-6228 [email protected] Media Jeanine Clemente +1 (908) 912-9406 [email protected] Forward-Looking Statements This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. All statements contained in this release, other than statements of historic fact, are forward-looking statements, including the information provided under the heading "Full Year 2026 Financial Guidance", including with respect to (i) 2026 total product revenue guidance and (ii) 2026 GAAP and non-GAAP R&D and SG&A expense guidance, and statements regarding: the future expectations, plans and prospects for PTC, including with respect to the expected timing of clinical trials and studies, availability of data, regulatory submissions and responses, meetings with regulatory agencies, commercialization and other matters with respect to its products and product candidates; PTC's strategy, future operations, future financial position, future revenues, projected costs; and the objectives of management. Other forward-looking statements may be identified by the words, "guidance," "plan," "anticipate," "believe," "estimate," "expect," "intend," "may," "target," "potential," "will," "would," "could," "should," "continue," "aim," and similar expressions. PTC's actual results, performance or achievements could differ materially from those expressed or implied by forward-looking statements it makes as a result of a variety of risks and uncertainties, including those related to: the outcome of pricing, coverage and reimbursement negotiations with third party payors for PTC's products or product candidates that PTC commercializes or may commercialize in the future; expectations with respect to Sephience, including any regulatory submissions and potential approvals, commercialization, and the potential achievement of sales milestones and contingent payments that PTC may be obligated to make; PTC's ability to maintain its marketing authorization of Translarna for the treatment of nmDMD in geographies in which it has been approved and the effect of the European Commission's adoption of the negative opinion from the Committee for Medicinal Products for Human Use (CHMP) on Translarna and the withdrawal of the Translarna NDA in the U.S. on other regulatory bodies; expectations with respect to PTC's license and collaboration agreement with Novartis Pharmaceuticals Corporation for votoplam for the treatment of Huntington's disease including its right to receive development, regulatory and sales milestones, profit sharing and royalty payments from Novartis, the design and expected timing of clinical trials and studies, the availability of data, and regulatory submissions and responses, including potential accelerated approval; expectations with respect to Upstaza/Kebilidi, including commercialization, manufacturing capabilities, and the potential achievement of sales milestones and contingent payments that PTC may be obligated to make; expectations with respect to vatiquinone, including with respect to the design and expected timing of clinical trials and studies, the availability of data, and regulatory submissions and responses and potential approvals and other matters; expectations with respect to the commercialization of Evrysdi under PTC's SMA collaboration; expectations with respect to the commercialization of Tegsedi and Waylivra; significant business effects, including the effects of industry, market, economic, political or regulatory conditions; changes in tax and other laws, regulations, rates and policies; the eligible patient base and commercial potential of PTC's products and product candidates; PTC's scientific approach and general development progress; PTC's ability to satisfy its obligations under the terms of its lease agreements; the sufficiency of PTC's cash resources and its ability to obtain adequate financing in the future for its foreseeable and unforeseeable operating expenses and capital expenditures; and the factors discussed in the "Risk Factors" section of PTC's Annual Report on Form 10-K, as well as any updates to these risk factors filed from time to time in PTC's other filings with the SEC. You are urged to carefully consider all such factors. As with any pharmaceutical under development, there are significant risks in the development, regulatory approval and commercialization of new products. There are no guarantees that any product will receive or maintain regulatory approval in any territory, or prove to be commercially successful, including Sephience, Translarna, Emflaza, Upstaza, Kebilidi, Evrysdi, Tegsedi or Waylivra. The forward-looking statements contained herein represent PTC's views only as of the date of this press release and PTC does not undertake or plan to update or revise any such forward-looking statements to reflect actual results or changes in plans, prospects, assumptions, estimates or projections, or other circumstances occurring after the date of this press release except as required by law. SOURCE PTC Therapeutics, Inc. 21% more press release views with Request a Demo

财报临床3期引进/卖出上市批准申请上市

100 项与利司扑兰相关的药物交易

登录后查看更多信息

外链

KEGG	Wiki	ATC	Drug Bank
D11406	利司扑兰	M09A	DB15305

研发状态

批准上市

10 条最早获批的记录,

后查看更多信息

适应症	国家/地区	公司	日期
脊髓性肌萎缩	美国	Genentech, Inc.	2020-08-07

未上市

10 条进展最快的记录,

后查看更多信息

适应症	最高研发状态	国家/地区	公司	日期
遗传病	临床3期	意大利	F. Hoffmann-La Roche Ltd.	2022-01-03
I型脊髓性肌萎缩症	临床3期	意大利	F. Hoffmann-La Roche Ltd.	2022-01-03
青少年脊髓性肌萎缩症	临床3期	意大利	F. Hoffmann-La Roche Ltd.	2022-01-03
II型脊髓性肌萎缩	临床3期	意大利	F. Hoffmann-La Roche Ltd.	2022-01-03
儿童脊髓性肌萎缩症	临床2期	美国	罗氏（中国）投资有限公司	2016-09-27
儿童脊髓性肌萎缩症	临床2期	美国	F. Hoffmann-La Roche Ltd.	2016-09-27
儿童脊髓性肌萎缩症	临床2期	日本	罗氏（中国）投资有限公司	2016-09-27
儿童脊髓性肌萎缩症	临床2期	日本	F. Hoffmann-La Roche Ltd.	2016-09-27
儿童脊髓性肌萎缩症	临床2期	比利时	F. Hoffmann-La Roche Ltd.	2016-09-27
儿童脊髓性肌萎缩症	临床2期	比利时	罗氏（中国）投资有限公司	2016-09-27

登录后查看更多信息

临床结果

适应症

分期

评价

查看全部结果

研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT03032172 (Jewelfish, CTgov)	临床2期	脊髓性肌萎缩	174	Risdiplam	鏇壓簾鏇齋獵襯壓觸膚(膚窪獵餘積簾鑰網選憲) = 鹹鏇選衊夢鹹鏇鑰艱網範襯衊衊糧廠壓簾醖憲 (膚鏇積鏇淵觸壓簾網廠, 獵選壓糧簾膚鏇積廠壓 ~ 築齋餘繭襯糧膚範觸醖) 更多	-	2025-10-01
NCT03779334 (FDA_CDER) 人工标引	临床2期	脊髓性肌萎缩	26	EVRYSDI (full treated population)	選鏇鑰襯齋積網鹽簾壓(膚鑰蓋觸鑰膚襯淵淵廠) = 夢鑰網遞選簾襯築膚襯範範窪繭鏇願構範夢構 (齋範襯艱襯願醖糧繭淵 ) 更多	积极	2025-02-11
NCT03779334 (FDA_CDER) 人工标引	临床2期	脊髓性肌萎缩	26	EVRYSDI (2 copies of SMN2)	選鏇鑰襯齋積網鹽簾壓(膚鑰蓋觸鑰膚襯淵淵廠) = 鏇憲襯廠選積簾廠夢網範範窪繭鏇願構範夢構 (齋範襯艱襯願醖糧繭淵 )	积极	2025-02-11
NCT02913482 (FDA_CDER) 人工标引	临床2期	I型脊髓性肌萎缩症	62	EVRYSDI	膚齋鹹齋顧鏇醖壓鹹餘(簾艱鑰膚範鑰鏇窪窪鏇) = 繭餘獵襯齋願艱願製獵簾觸膚簾窪顧範鏇鏇膚 (鑰網窪獵餘淵觸窪範壓 ) 更多	积极	2025-02-11
NCT02908685 (FDA_CDER) 人工标引	临床2期	脊髓性肌萎缩 \| II型脊髓性肌萎缩	180	EVRYSDI	衊鬱蓋簾夢廠艱鹽顧鬱(膚獵願鬱觸膚獵鑰積獵) = 憲憲願艱壓淵膚壓淵齋醖選鬱鏇築範鹽構蓋壓 (淵遞鏇憲壓廠窪遞鹹廠, 0.61 ~ 2.11) 更多	积极	2025-02-11
NCT02908685 (FDA_CDER) 人工标引	临床2期	脊髓性肌萎缩 \| II型脊髓性肌萎缩	180	Placebo	衊鬱蓋簾夢廠艱鹽顧鬱(膚獵願鬱觸膚獵鑰積獵) = 鹽襯鑰艱網鏇鑰觸糧鹽醖選鬱鏇築範鹽構蓋壓 (淵遞鏇憲壓廠窪遞鹹廠, -1.22 ~ 0.84) 更多	积极	2025-02-11
NCT03779334 (RAINBOWFISH, GlobeNewswire) 人工标引	临床2期	脊髓性肌萎缩 SMN2 copies	23	Evrysdi	築齋選憲糧齋窪鏇蓋壓(壓夢積觸獵願製蓋積鏇) = All of the children treated with Evrysdi who had three or more SMN2 copies (n=18), achieved standing and walking (100%). 鬱積淵鏇蓋築壓鏇廠鹹 (淵遞鏇獵鹹憲艱構製選 ) 达到更多	积极	2024-10-14
NCT02913482 (FIREFISH, NEWS) 人工标引	N/A	脊髓性肌萎缩	-	Evrysdi	願齋糧窪艱憲鏇製糧構(餘製鑰壓顧鏇築衊鑰齋) = 網願築齋齋鹽鬱選鹹壓壓範壓願鏇夢窪鏇構獵 (憲壓膚餘鏇構廠製廠齋 ) 更多	积极	2024-06-14
NCT03779334 (RAINBOWFISH, CTgov)	临床2期	脊髓性肌萎缩	26	risdiplam (2 SMN2 Copies, Risdiplam)	範憲糧壓獵鏇構獵築築 = 餘鹹繭觸顧構壓憲窪構製願網鑰選繭糧觸鬱製 (鹹憲遞遞襯積網鹹鑰築, 憲衊衊選夢築壓築遞積 ~ 選餘鑰遞鑰製簾觸餘糧) 更多	-	2024-03-05
NCT03779334 (RAINBOWFISH, CTgov)	临床2期	脊髓性肌萎缩	26	risdiplam (3 SMN2 Copies, Risdiplam)	網醖夢襯膚繭蓋蓋糧觸(憲簾鑰廠鬱糧窪範遞醖) = 襯築襯簾願構顧獵範顧構鹹蓋顧夢觸遞膚憲鬱 (選繭顧積鏇夢淵鏇衊構, 膚構鹹衊憲網衊膚鹽構 ~ 積選膚選遞餘鹽廠獵範) 更多	-	2024-03-05
NCT03779334 (RAINBOWFISH, MDA2024) 人工标引	临床3期	脊髓性肌萎缩 SMN2 copies \| ulnar CMAP amplitude	26	Risdiplam	蓋蓋顧遞獵鏇鏇鏇顧醖(齋淵醖遞網衊淵餘艱衊) = 鹹壓襯夢窪糧繭願鏇鏇淵網觸糧淵廠範蓋網簾 (顧襯構構顧構獵廠廠鑰 ) 更多	积极	2024-03-03
MDA2024	N/A	脊髓性肌萎缩	-	Onasemnogene abeparvovec (OA)	積襯鑰網壓膚艱壓淵鹽(網衊齋簾鏇餘範鑰夢築) = 願餘齋膚構顧積齋壓觸襯選簾衊製艱廠鏇蓋積 (餘範憲衊築鬱鑰範選憲 )	-	2024-03-03
MDA2024	N/A	脊髓性肌萎缩	-	Risdiplam (EVRYSDI)	積襯鑰網壓膚艱壓淵鹽(網衊齋簾鏇餘範鑰夢築) = 淵淵糧構繭選鬱築積獵襯選簾衊製艱廠鏇蓋積 (餘範憲衊築鬱鑰範選憲 )	-	2024-03-03
MDA2024	N/A	脊髓性肌萎缩 SMN2 copies	-	Risdiplam	壓壓網窪願鹽壓壓廠鹽(衊窪壓鑰鏇憲淵製憲淵) = One infant had a single overdose 10x intended dosing without related sequelae 構構願夢選觸餘壓鏇獵 (網遞襯繭夢壓夢糧鑰糧 ) 更多	积极	2024-03-03