诺西那生钠(Nusinersen sodium) - 药物靶点：SMN2_在研适应症：脊髓性肌萎缩_专利_临床

概要

基本信息

药物类型

ASO

别名

IONIS SMN Rx、IONIS-SMNRx、ISIS SMNRx

+ [14]

靶点

SMN2

作用方式

刺激剂

作用机制

SMN2刺激剂(运动神经元存活蛋白刺激剂)、RNA干扰

治疗领域

神经系统疾病其他疾病

在研适应症

脊髓性肌萎缩

非在研适应症

I型脊髓性肌萎缩症

原研机构

Ionis Pharmaceuticals, Inc.

在研机构

Biogen, Inc.

渤健生物科技（上海）有限公司Biogen Idec Research Ltd.

+ [4]

非在研机构-

权益机构

Ionis Pharmaceuticals, Inc.

Royalty Pharma Plc

Biogen, Inc.

最高研发阶段批准上市

首次获批日期

美国 (2016-12-23),

脊髓性肌萎缩

最高研发阶段(中国)批准上市

特殊审评孤儿药 (美国)、孤儿药 (日本)、孤儿药 (韩国)、临床急需境外新药 (中国)、快速通道 (美国)

登录后查看时间轴

结构/序列

使用我们的RNA技术数据为新药研发加速。

登录

或

查看完整样例数据

Sequence Code 29854605

来源: *****

关联

27

项与诺西那生钠相关的临床试验

NCT07047144

/ Recruiting临床2期

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.

NCT06555419

/ Recruiting临床1期

An Open Label, Single Cohort Study to Assess the Pharmacokinetic Profile of Nusinersen (BIIB058) Administered Via the ThecaFlex DRx™ System (PIERRE-PK)

In this PIERRE-PK study, researchers will learn how the body processes nusinersen when it is given through the ThecaFlex DRx™ System, compared to when nusinersen is given by lumbar puncture (LP). The ThecaFlex DRx system is an investigational implantable medical device developed by Alcyone Therapeutics, Inc. It consists of a catheter, which is a flexible tube, connected to a port which is placed under the skin. Alcyone Therapeutics, Inc. has an ongoing study called PIERRE to test the ThecaFlex DRx system. Participants with spinal muscular atrophy (SMA) in the PIERRE study may be enrolled in the PIERRE-PK study.
The main objective of the PIERRE-PK study is to learn how the body processes nusinersen when given by the ThecaFlex DRx system compared to a lumbar puncture. The main questions researchers want to answer are:
* What is the highest amount of nusinersen found in the blood after dosing?
* How much nusinersen is found in the blood over the first 24 hours after dosing?
The PIERRE-PK study will be done as follows:
* Participants will be screened to check if they can join the study. The screening period will be up to 30 days for this study and may overlap with the PIERRE study.
* Participants will receive a dose of nusinersen by lumbar puncture.
* The ThecaFlex DRx system will be implanted after the lumbar puncture, as part of the PIERRE study.
* Participants will receive a dose of nusinersen by the ThecaFlex DRx system, as part of the PIERRE study.
* Researchers will take blood samples before and after each dose. The last blood sample will be taken 24 hours after the dose.
* The total study duration for each participant in the PIERRE-PK study will be approximately 5 months. This period will overlap with the participant's first 5 months in the PIERRE study.

开始日期2025-01-16

申办/合作机构

Biogen, Inc.

[+1]

ACTRN12626000157370

/ Active, not recruitingN/AIIT

Assessment of Functionality of Patients with Type IV Spinal Muscular Atrophy Treated with Spinraza: A Study with use of the Timed Up and Go Test, the Six-Minute Walk Test, the Hammersmith Functional Motor Scale, and Stabilometric Platform Test

开始日期2024-04-30

申办/合作机构-

100 项与诺西那生钠相关的临床结果

登录后查看更多信息

100 项与诺西那生钠相关的转化医学

登录后查看更多信息

100 项与诺西那生钠相关的专利（医药）

登录后查看更多信息

765

项与诺西那生钠相关的文献（医药）

2026-04-01·BRAIN & DEVELOPMENT

Outcomes of combination therapy with nusinersen, onasemnogene abeparvovec, and risdiplam over 3.5 years in a patient with prenatally diagnosed spinal muscular atrophy type 0: A case report

Article

作者: Saito, Kayoko ; Yokomura, Mamoru ; Togashi, Noriko ; Yagi, Yuki ; Haginoya, Kazuhiro ; Otsuki, Noriko ; Katata, Yu ; Okubo, Yukimune ; Endo, Wakaba ; Morishita, Yuko ; Kato, Tamaki ; Kawaji, Takahiro ; Inui, Takehiko ; Uneoka, Saki ; Kawashima, Aritomo ; Yoshida, Sayaka

BACKGROUND:

Recently introduced disease-modifying therapies have markedly improved survival and motor outcomes, particularly when initiated presymptomatically or in the early symptomatic stage. However, reports on treatment of the most severe form, SMA type 0, remain extremely limited.

CASE REPORT:

We report a patient with prenatally diagnosed spinal muscular atrophy type 0 who was treated with nusinersen and onasemnogene abeparvovec as early and as safely as possible, followed by risdiplam, for further motor acquisition. With continuous physiotherapy, the patient achieved independent walking at 2 years 11 months and showed sustained development without swallowing or respiratory difficulties up to 3.5 years of age.

DISCUSSION:

Early intervention with combined use of disease-modifying therapies may be beneficial for patients with severe spinal muscular atrophy phenotypes. However, since evidence of efficacy and safety of combination therapy is still lacking, great caution should be paid to this treatment strategy, taking the patient's clinical status into account.

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 ; Moggio, Maurizio ; Panicucci, Chiara ; 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.

1,162

项与诺西那生钠相关的新闻（医药）

2026-04-01

·生物经纬

药企药品周讯 | 首款且唯一！每周一次的长效基础胰岛素获批上市

01 3月27日，诺和诺德宣布美国食品药品监督管理局（FDA）已批准Awiqli®（依柯胰岛素注射液，700单位/毫升）上市。该药物是首款且唯一一款每周一次的长效基础胰岛素，适用于作为饮食和运动的辅助手段，改善成人2型糖尿病患者的血糖控制。 02 当地时间3月30日，美国FDA批准渤健和 Ionis联合开发的诺西那生（Spinraza）高剂量（50 mg/28 mg）方案上市，用于治疗脊髓性肌萎缩症（SMA）。此前 FDA 已批准诺西那生 12 mg治疗方案。2025年4月，高剂量诺西那生治疗方案也已在中国报上市。 03 3月30日，阿斯利康宣布，中国 NMPA已正式批准科赛优®（硫酸氢司美替尼胶囊）适应症拓展，用于3岁及3岁以上伴有症状、无法手术的丛状神经纤维瘤（PN）的1型神经纤维瘤病（NF1）儿童及成人患者的治疗。此前，司美替尼仅在国内获批用于该适应症的儿童患者，本次获批使其成为国内首个且唯一获批用于3岁及3岁以上NF1-PN患者的治疗药物。 04 3月27日，NMPA官网显示，阿斯利康和安进联合递交的 TSLP单抗特泽利尤单抗注射液获批上市。本次该药获批2个适应症，分别是哮喘和慢性鼻窦炎伴鼻息肉病。 05 3月19日，药监局批准三生制药申报的罗赛促红素α注射液（商品名：新比澳）上市，该品种用于治疗因慢性肾脏病引起的贫血，且正在接受促红细胞生成素治疗的血液透析患者。 06 当地时间3月19日，葛兰素史克（GSK）宣布，美国FDA已批准利奈昔巴特（Linerixibat）上市，用于治疗原发性胆汁性胆管炎（PBC）成人患者的胆汁淤积性瘙痒。 07 3月18日，诺华公司宣布，「司库奇尤单抗」新适应症获得美国FDA批准，适用于12岁及以上儿童中重度化脓性汗腺炎（HS）患者的治疗。司库奇尤单抗是全球首个获批的全人源白介素（IL）-17A抑制剂，可特异性中和多种来源的IL-17A，抑制其促炎作用。 01 3月30日，百奥赛图（北京）医药科技股份有限公司（以下简称“百奥赛图”），一家以创新技术驱动新药研发的国际性生物技术公司，今日宣布与大正制药株式会社（以下简称“大正制药”）达成一项平台授权协议。百奥赛图授予大正制药其专有的RenNano®全人仅重链抗体（HCAb）发现平台的使用权，以支持大正制药的内部新药研发项目。 02 3月29日，由生成式人工智能（AI）驱动的生物医药科技公司英矽智能（Insilico Medicine, 3696.HK），宣布与礼来（Lilly）达成药物发现合作，这项合作将利用英矽智能的AI引擎加速多个治疗领域中新型疗法的发现与开发。这项由人工智能驱动的合作包含一项全球独家许可，涵盖涉及多个治疗领域的系列项目。英矽智能将有资格获得1.15亿美元首付款，并在后续达成开发、监管及商业化里程碑后获得进一步付款，使交易总价值最高可达约27.5亿美元。由于交易规模庞大，触发了美国联邦贸易委员会（FTC）的反垄断审查。 03 3月27日，上海华奥泰生物药业股份有限公司（简称“华奥泰”）宣布已与Almirall, S.A.（简称“Almirall”）就一款早期单克隆抗体候选药物达成全球研究合作及许可协议，开发具有多种潜在适应症（包括医学皮肤科）的新靶点的单克隆抗体候选药物。根据协议，华奥泰拥有在中国开发和商业化项目和产品的权利，Almirall拥有在中国以外的全球开发和商业化项目和产品的权利。利用创新研发平台，华奥泰将开展相关研究，直至获得具有临床概念性验证的分子，Almirall将向华奥泰支付总额3.4亿美元的首付款、开发和商业化里程碑付款，以及相应的分级特许权使用费。 04 3月31日，Biogen宣布以56亿美元收购Apellis Pharmaceuticals（Nasdaq:APLS），根据该协议，Biogen同意在收盘时以每股41.00美元的现金或约56亿美元的价格收购Apellis的所有已发行股票。Apellis是推进严重补体驱动疾病治疗的领导者，其加入有望通过在其增长组合中增加两种商业化的差异化免疫学和罕见病药物来增强Biogen的短期和长期收入增长。EMPAVELI®和SYFOVRE®为Biogen增加了即时销售收入，2025年的净销售额合计为6.89亿美元，预计至少到2028年，这一数字将以百分之十几的速度增长。 05 3月31日，礼来宣布以总额78亿美元收购美股上市公司Centessa Pharmaceuticals。前期现金对价代表了约63亿美元的总股权价值，CVR代表了约15亿美元的额外潜在总股权价值。该交易将根据英格兰和威尔士法律通过安排方案实现，预计将在第三季度完成。Centessa正在推进一系列食欲素受体2（OX2R）激动剂，旨在解决对睡眠-觉醒周期至关重要的神经生物学系统，以治疗白天过度嗜睡和觉醒障碍。 06 3月27日，东京和纽约，Otsuka（大冢制药）和Transcend Therapeutics宣布两家公司签署协议，根据协议大冢制药将通过其全资子公司Otsuka America全面收购Transcend。Transcend成立于2021年，是一家临床阶段的生物技术公司，致力于开发神经精神疾病的快速治疗方法。该公司正在开发的TSND-201（甲基酮/methylone）是一种快速作用的神经弹性原，一种在大脑中诱导快速持久神经可塑性的药物，目前正被作为创伤后应激障碍（PTSD）和其他精神疾病的潜在治疗方法。 07 3月27日，巴塞尔，诺华公司宣布已达成协议收购Excellergy，这是一家私人生物技术公司，正在开发针对IgE驱动疾病的下一代抗IgE疗法。拟议的收购获得Exl-111，一款半衰期延长的高亲和力抗IgE抗体。根据协议条款，诺华将支付高达20亿美元的预付款和里程碑式付款来收购Excellergy。往期回顾药企药品周讯 | 首款EGFR×HER3双抗ADC“iza-bren”在华申报上市药企药品周讯 | 1分钟完成注射！罗氏莫妥珠单抗皮下注射制剂获批上市药企药品周讯 | 重磅！血液新药卡拉西珠单抗国内获批药企药品周讯 | 重要突破！国产HIV1类新药首次获批临床药企药品周讯 | 疾病控制率达100%！康方生物双抗新药治疗三阴性乳腺癌拟纳入突破性治疗品种综合整理 | 王超，贺银慧（NO.803期）图文编辑 | 王小雨商务合作丨马经理 15850522742 新闻投稿/访谈邀约彭经理 18012921198

上市批准CSCO会议

2026-04-01

·DrG的雍熙路

Elevar抗癌小分子lirafugratinib获FDA优先审评资格，用于胆管癌；渤健Spinraza高剂量方案获FDA批准，用于脊髓性肌萎缩症

行业资讯 Elevar抗癌小分子lirafugratinib获FDA优先审评资格，用于胆管癌近日，Elevar Therapeutics宣布其FGFR2抑制剂lirafugratinib的新药申请获FDA受理并授予优先审评，用于既往治疗失败且携带FGFR2融合或重排的胆管癌患者，预计将于2026年9月作出审评决定。该资格基于ReFocus I/II期研究，数据显示其确认客观缓解率达46.5%，安全性总体可控。作为高选择性口服小分子，该药在多项研究中展现出良好的抗肿瘤活性。渤健Spinraza高剂量方案获FDA批准，用于脊髓性肌萎缩症近日，渤健宣布FDA批准其反义寡核苷酸疗法Spinraza高剂量给药方案，用于脊髓性肌萎缩症治疗。该方案包含50 mg和28 mg剂量，较既往12 mg方案提高药物暴露。批准基于DEVOTE II/III期研究，在未治疗症状性婴儿中显著改善运动功能，CHOP-INTEND评分较对照提升26.19分（p<0.0001）。Spinraza通过调控SMN2剪接增加SMN蛋白表达，此次获批有望进一步提升临床获益。元思生肽完成数亿美元B轮融资，加码智能化大环肽研发平台 3月，元思生肽宣布完成数亿美元B轮融资，由国际生物科技基金领投，德诚资本与鼎晖VGC联合领投，多家主权基金及产业资本跟投，原有股东持续加持。募集资金将用于升级Synova™大环肽研发平台并推进多条管线进入临床。公司聚焦肿瘤、自身免疫及代谢疾病等领域，布局口服环肽、细胞渗透性肽及多肽偶联药物，加速创新药研发与商业化进程。学术前沿 Memorial Sloan Kettering团队在Cell 报道uPAR靶向CAR-T开辟实体瘤新路径 3月30日，美国Memorial Sloan Kettering Cancer Center研究团队在期刊Cell 发表最新研究发现，细胞表面蛋白uPAR可标记肿瘤及其支持性成纤维细胞、免疫抑制性髓系细胞等“高致瘤状态”，构成肿瘤生态系统。基于此，团队开发uPAR靶向CAR-T细胞，可同时清除肿瘤及微环境，显著抑制卵巢癌、肺癌、胰腺癌等实体瘤，并在临床前模型中显示有限正常组织毒性。化疗可诱导uPAR上调，为CAR-T识别提供增强靶点，实现“诱导+清除”的系统性治疗策略。北大团队在Nature Communications 揭示STAT6新机制延缓免疫衰老个体衰老常伴随免疫失调及炎性衰老进程。北京大学、北大-清华生命科学联合中心邱义福团队联合北京大学第三医院孙永昌团队在Nature Communications 发表研究，揭示DNA-PK/STAT6/PU.1分子轴在巨噬细胞抗衰中的关键作用。研究发现，DNA损伤可诱导DNA-PK介导STAT6 Ser807位点磷酸化，稳定其蛋白并增强DNA修复基因转录，从而延缓细胞衰老。该机制依赖巨噬细胞特异性转录因子PU.1协同作用。动物模型显示，该通路可改善肺纤维化表型，在慢阻肺患者中亦呈保护相关性，为干预炎性衰老提供潜在靶点。 AI点评本组进展体现“精准靶向深化+剂量优化+免疫与衰老机制突破”。FGFR2抑制剂获优先审评，强化胆管癌分子分型治疗；Spinraza高剂量方案提升SMA临床获益；大环肽平台融资加速新型分子形态布局；uPAR CAR-T拓展实体瘤微环境协同清除策略；而STAT6相关机制则为免疫衰老与慢病干预提供新靶点。整体创新正向更精准与系统性调控演进。参考信源： 1.https://mp.weixin.qq.com/s/m8TCaJvRM3LVR49rD2n8rA 2.https://mp.weixin.qq.com/s/m8TCaJvRM3LVR49rD2n8rA 3.https://mp.weixin.qq.com/s/8CEDOa7p-gvsJ4PGnGE9xw 4.https://mp.weixin.qq.com/s/LysI3gSCHUq64k2eSXwaxA 5.https://mp.weixin.qq.com/s/1BOzI624QY_xUnIWQVHn8w 免责声明 :本文所含的信息来源于文中可靠信源，仅供读者一般参考之用，不应作为决策依据，DrG的雍熙路对因使用本文内容而造成的一切损失，不承担任何责任。部分图片来源于网络，如涉及版权问题，敬请联系我们以便采相应处理举措。我们鼓励转发分享及合理引用本文内容，但请在引用时于明显位置标注文章来源。

2026-04-01

·PRNewswire

FDA Approves High Dose Regimen of SPINRAZA for People Living with SMA

March 30, 2026 -- Cure SMA, the leading nonprofit organization supporting those impacted by spinal muscular atrophy (SMA), celebrates that Biogen today announced FDA approval of the High Dose regimen of SPINRAZA™ (nusinersen) to treat people living with SMA. With broad label approval for all those living with SMA, this is an excellent result for the SMA community. SPINRAZA is an antisense oligonucleotide therapy that increases production of survival motor neuron (SMN) protein, essential for motor neuron function. The High Dose regimen is designed to further enhance SMN protein levels. "The FDA approval of the High Dose regimen of SPINRAZA is a major advancement for our community," said Kenneth Hobby, President at Cure SMA. "This new regimen offers hope for even stronger outcomes and more individualized care for people living with SMA. It's a testament to decades of research, clinical trial participation, and the dedication of individuals and families, industry and healthcare professionals who have driven progress in SMA treatment." Cure SMA will host a community webinar on April 30, 2026, to provide education and guidance on the High Dose regimen of SPINRAZA option, including next steps for ensuring broad access, and practical resources to support individuals and families in making informed decisions with their healthcare teams. "With this approval, our focus turns to ensuring individuals and families living with SMA understand what this means and making sure they have a clear path to access," said Jackie Glascock, PhD, Chief Scientific Officer at Cure SMA. "We are committed to partnering with Biogen and healthcare providers to support education, access, and implementation so patients can benefit fully from this High Dose regimen of SPINRAZA option." Cure SMA extends its sincere gratitude to individuals living with SMA, as well as to the donors, advocates, healthcare professionals, researchers, and clinical trial participants whose collective efforts have made this milestone possible. Over the past 30 years, Cure SMA has invested more than $92 million in SMA research, helping to build the scientific foundation for therapies such as the High Dose regimen of SPINRAZA. Participation in clinical trials and ongoing research contributions have been instrumental in generating the data that supported this approval and advancing treatment options for the SMA community. Cure SMA also recognizes the continued commitment of Biogen in expanding therapeutic options for individuals with SMA and extends its appreciation to the U.S. Food and Drug Administration for its ongoing collaboration and attention to the unmet needs of the SMA community. Spinal muscular atrophy (SMA) is a progressive neurodegenerative disease affecting motor nerve cells in the spinal cord and the muscles used for breathing, eating, crawling, and walking. SMA affects approximately one in 15,000 births in the U.S., and about one in every 50 Americans is a genetic carrier. Cure SMA is the leading nonprofit organization driving research, advocacy, and support for individuals and families living with SMA. Its mission is to advance research for treatments and a cure, and to support and empower everyone impacted by SMA today. The content above comes from the network. if any infringement, please contact us to modify.

上市批准寡核苷酸

100 项与诺西那生钠相关的药物交易

登录后查看更多信息

外链

KEGG	Wiki	ATC	Drug Bank
D10791	诺西那生钠	M09AX07	DB13161

研发状态

批准上市

10 条最早获批的记录,

登录

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	Biogen, Inc.	2016-12-23

未上市

10 条进展最快的记录,

登录

后查看更多信息

适应症	最高研发状态	国家/地区	公司	日期
I型脊髓性肌萎缩症	临床3期	美国	Biogen, Inc.	2014-08-19
I型脊髓性肌萎缩症	临床3期	日本	Biogen, Inc.	2014-08-19
I型脊髓性肌萎缩症	临床3期	澳大利亚	Biogen, Inc.	2014-08-19
I型脊髓性肌萎缩症	临床3期	比利时	Biogen, Inc.	2014-08-19
I型脊髓性肌萎缩症	临床3期	加拿大	Biogen, Inc.	2014-08-19
I型脊髓性肌萎缩症	临床3期	法国	Biogen, Inc.	2014-08-19
I型脊髓性肌萎缩症	临床3期	德国	Biogen, Inc.	2014-08-19
I型脊髓性肌萎缩症	临床3期	意大利	Biogen, Inc.	2014-08-19
I型脊髓性肌萎缩症	临床3期	韩国	Biogen, Inc.	2014-08-19
I型脊髓性肌萎缩症	临床3期	西班牙	Biogen, Inc.	2014-08-19

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


NCT02386553 (NURTURE, CTgov)	临床2期	脊髓性肌萎缩	25	nusinersen (ISIS 396443 2 SMN2 Copies)	齋願餘餘網遞獵願蓋糧(蓋範膚鑰簾構齋淵襯淵) = 膚築餘壓鑰獵餘範構艱衊簾獵繭繭鑰淵醖選範 (壓醖夢餘觸壓範遞鬱簾, 繭淵夢窪壓鹹鏇衊簾餘 ~ 觸繭繭繭淵鑰鏇鹽製艱) 更多	-	2025-10-20
				nusinersen (ISIS 396443 3 SMN2 Copies)	構襯選網蓋鏇選鏇醖壓 = 餘壓鏇遞簾願鏇選鏇蓋艱簾獵醖糧構觸廠齋製 (顧網鑰鑰蓋遞糧鏇夢壓, 築憲衊襯積鹽窪築選鬱 ~ 獵範窪衊齋憲艱鑰夢鏇) 更多
NCT02386553 (NURTURE, Company_Website) 人工标引	临床2期	脊髓性肌萎缩	25	SPINRAZA 12mg	糧範醖範鑰鬱網鑰淵顧(網鹹窪餘衊獵膚廠夢構) = 襯鏇積觸夢築襯製齋觸窪製廠襯衊憲壓繭衊構 (壓遞獵範築繭糧蓋壓選 ) 更多	积极	2025-06-27
NCT04089566 (DEVOTE, Company_Website) 人工标引	临床3期	脊髓性肌萎缩	38	Nusinersen (received one loading dose (50 mg) and two maintenance doses (28 mg each))	獵淵積鹽築選鬱廠鹹襯(鹹醖積鹹襯鹽淵廠淵糧) = 範齋鹹鬱製衊獵範築鏇醖廠蓋餘鹽選夢膚積製 (築範網獵夢壓淵艱鏇蓋, 0.49 ~ 4.56) 更多	积极	2025-06-27
NCT04089566 (DEVOTE, MDA2025) 人工标引	临床2/3期	脊髓性肌萎缩	145	nusinersen 50/28mgnusinersen 50/28mg	糧鏇鑰願鹽夢膚衊鹹獵(鏇醖顧膚鑰壓築鏇壓範) = 衊衊鬱構醖製鹹鏇憲積顧鹽觸構鑰襯餘鏇鏇築 (繭廠製獵衊艱夢繭範鏇 ) 更多	积极	2025-03-16
				nusinersen 12/12mgnusinersen 12/12mg	觸觸獵構遞範壓襯壓艱(鏇膚選窪繭壓糧鏇蓋鹹) = 觸蓋襯觸簾獵製淵夢艱廠蓋衊衊繭選願糧願鬱 (製餘鹽膚艱夢願築網憲 ) 更多
NCT04488133 (RESPOND, MDA2025)	临床2期	脊髓性肌萎缩	46	Nusinersen (age ≤9 mo with 2 SMN2 copies)	餘齋憲艱艱鬱製構齋夢(膚遞獵鏇網艱鏇網廠廠) = Three (7%) children had a nusinersen-related mild AE (proteinuria). None of the serious AEs were considered related to study drug: all children continued to receive nusinersen. 鏇醖憲衊憲獵製遞鬱遞 (積餘廠鹹廠窪鹽壓製網 )	积极	2025-03-16
				Nusinersen (age >9 mo with 2 copies)
NCT02594124 (SHINE Study (1692) \| SHINE, CTgov)	临床3期	脊髓性肌萎缩	292	nusinersen (Infantile SMA Onset CS3A)	糧繭選壓憲醖衊鑰顧製 = 糧構觸網蓋觸憲鑰餘醖構範窪窪構積鏇蓋糧餘 (壓鹹選餘繭艱蓋壓齋網, 鑰糧選範鏇餘蓋夢醖憲 ~ 鹽顧製繭夢遞醖廠餘窪) 更多	-	2024-10-22
				nusinersen (Infantile SMA Onset CS3B Previous Control)	糧繭選壓憲醖衊鑰顧製 = 淵簾鹹壓築願築膚廠淵構範窪窪構積鏇蓋糧餘 (壓鹹選餘繭艱蓋壓齋網, 網壓構鏇鑰鹽願襯齋顧 ~ 蓋憲憲淵遞鑰廠餘鏇壓) 更多
NCT04089566 (DEVOTE, NEWS) 人工标引	临床2/3期	脊髓性肌萎缩	75	Spinraza 50/28 mg Active Treatment Group	鬱膚觸鹽築鏇獵廠鑰鹹(願膚衊製遞觸襯憲築壓) = Biogen on Wednesday said that an experimental, higher dose of its spinal muscular atrophy drug Spinraza met the primary endpoint of a clinical trial comparing it with a historical control group. 蓋築廠餘壓淵獵積選艱 (選廠簾鏇淵積齋衊鹽壓 ) 达到更多	积极	2024-09-04
				Spinraza 28/28 Milligram (mg) Safety Group
NCT04419233 (PANDA, Pubmed \| Adv Ther)	临床4期	脊髓性肌萎缩	50	Nusinersen	齋壓淵衊糧窪網窪淵網(鹹鬱糧齋艱艱積憲範築) = 壓艱獵夢膚積繭廠襯襯窪餘鹽艱鹹窪廠醖鹹選 (餘鏇壓繭廠餘鏇顧築遞 )	积极	2024-07-01
NCT04488133 (RESPOND, GlobeNewswire) 人工标引	临床4期	脊髓性肌萎缩 Nfl	29	SPINRAZA	簾鹹網艱醖鏇製簾積築(膚範願鏇鏇窪鏇鏇顧餘) = 積鑰齋餘願蓋簾製觸衊齋壓壓鑰窪願鑰齋齋壓 (蓋築憲遞遞鬱獵餘製範 ) 更多	不佳	2024-03-06
NCT02386553 (NURTURE, MDA2023) 人工标引	临床2期	脊髓性肌萎缩 SMN2 copies	15	Nusinersen (2 SMN2 copies)	壓衊壓廠夢淵窪願鬱窪(鏇淵憲鹹鏇觸淵遞範網) = 壓願餘醖遞鹽衊衊鏇網觸衊願齋醖廠鏇構憲淵 (夢積膚繭糧鹹齋鏇夢遞 ) 更多	积极	2023-03-19
				Nusinersen ( 2 SMN2 copies who did not have CMAP <2 mV )	壓衊壓廠夢淵窪願鬱窪(鏇淵憲鹹鏇觸淵遞範網) = 膚膚憲觸鹹廠獵鑰獵製觸衊願齋醖廠鏇構憲淵 (夢積膚繭糧鹹齋鏇夢遞 ) 更多