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2025-09-01

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]

NCT06978985

/ RecruitingN/AIIT

Adult SMA REACH: Development and Implementation of a Standardised Data Set and Data Collection Research Study in the UK Adult SMA Population

Adult SMA REACH is a data collection study aiming to gain a better understanding of the impact of standards of care and new treatments on the natural history of Spinal Muscular Atrophy (SMA). This study is sponsored by The Newcastle upon Tyne Hospitals NHS Foundation Trust. Adult SMA REACH is funded by Biogen and Roche.
Currently, there are three drug treatments available for SMA in the UK: Zolgensma, Nusinersen and Risdiplam. Zolgensma is the only approved drug - Nusinersen and Risdiplam are currently available as part of Managed Access Agreements (MAA).

开始日期2022-07-29

申办/合作机构

The Newcastle upon Tyne Hospitals NHS Foundation Trust

[+3]

100 项与诺西那生钠相关的临床结果

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100 项与诺西那生钠相关的转化医学

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100 项与诺西那生钠相关的专利（医药）

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853

项与诺西那生钠相关的文献（医药）

2025-12-31·ANNALS OF MEDICINE

Unlocking the potential: advancements and applications of gene therapy in severe disorders

Review

作者: M. Elossaily, Gehan ; Makhijani, Shivani ; Ansari, Mohd Nazam ; Ingle, Rahul G.

INTRODUCTION:

Several severe disorders, such as inherited diseases (e.g. cystic fibrosis and beta thalassemia), genetic diseases (e.g. malignant tumors and diabetes), and infectious diseases (e.g. HIV) are pose significant challenges to human health.

BACKGROUND:

Over the past few decades, researchers have been working on gene therapies, and currently, terrible dreams have come true. To date, the Food and Drug Administration (FDA) has approved multiple gene therapies such as Kynamro for familial hypercholesterolaemia, Exondys51 for duchenne muscular dystrophy, Spinraza for spinal muscular atrophy, etc., rest for cancer, infectious diseases, and rare diseases.

DISCUSSION:

The authors have summarized recent advances in gene therapy, its background, molecular basis (e.g. viral and non-viral vectors), gene-editing techniques (e.g. CRISPR/Cas9, TALEN, ZFN), and its foremost applications in severe disorders, such as cancer, monogenic disorders (e.g. spinal muscular atrophy), polygenic disorders (e.g. autism), neurogenic disorders (e.g. Parkinson disease and Alzheimer's disease), and infectious diseases (e.g. HIV).

CHALLENGES:

In addition, we explored the major challenges faced by gene therapies during targeted delivery, immunogenicity, efficacy, and safety.

CONCLUSION:

To date, most of the promising approaches, such as different vectors, target cell populations, and both in vivo and ex vivo have paved the foundation for applications of gene therapies. Additionally, advances in enhancing the immune system that would certainly lower the healthcare costs. This review highlights the translatory potential of gene therapy in revolutionizing the treatment landscape for severe disorders.

2025-10-01·CLINICAL NEUROLOGY AND NEUROSURGERY

Surgical success of subcutaneous systems for intrathecal nusinersen delivery in spinal muscular atrophy: A systematic review and meta-analysis

Review

作者: Lu, Victor M ; Roach, Caleigh S ; Vazquez, Sima ; Niazi, Toba N

BACKGROUND:

The intrathecal administration of nusinersen to treat spinal muscular atrophy (SMA) has demonstrated therapeutic effect, however, repeat lumbar punctures confer multiple disadvantages. As such, subcutaneous systems have been described to provide an alternative, less invasive strategy for repeat administration. Correspondingly this study aimed to aggregate metadata to better define the surgical success of these subcutaneous systems.

METHODS:

Searches of multiple electronic databases from inception to March 2025 were conducted following PRISMA guidelines. Articles were screened against pre-specified criteria. Quantitative outcomes were pooled by random-effects meta-analyses where possible.

RESULTS:

There were 8 studies describing 74 SMA patients that were managed with a subcutaneous system for intrathecal nusinersen. No official system currently exists for this purpose, and all studies reported off-label use of either an intravascular port (5 of 8 studies) or reservoir system (3 of 8 studies) as the subcutaneous port. These ports were located mostly in the lateral flank (5 of 8 studies). In terms of intrathecal access, a hemilaminotomy was used universally in most studies (6 of 8 studies), and access was obtained most commonly in the thoracolumbar region (3 of 8 studies). A total of 22 individual adverse events were reported amongst the cohort, the most common being cerebrospinal fluid leak needing intervention (7/74, 9 %). The pooled estimate by meta-analysis indicated an overall weighted rate of adverse events to be 27 % (95 % CI 16-40 %). Eight patients required revision surgery, with a pooled revision surgery rate estimate of 9 % (95 % CI 2-19 %).

CONCLUSIONS:

Subcutaneous systems can be used in the setting of SMA for repeat nusinersen administration. There exists no consensus system or surgical approach currently, with multiple systems, port types, port location, intrathecal access techniques and intrathecal access locations reported. Future multidisciplinary efforts are needed to elevate the concept of subcutaneous delivery systems for SMA towards convention.

2025-10-01·NEUROBIOLOGY OF DISEASE

Nusinersen corrects L-arginine deficiency in the cerebrospinal fluid of patients with severe spinal muscular atrophy

Article

作者: Nuzzo, Tommaso ; D'Amico, Adele ; Usiello, Alessandro ; Caretto, Anna ; Bertini, Enrico ; Panicucci, Chiara ; Errico, Francesco ; Boido, Marina ; Hassan, Amber ; Pellizzoni, Livio ; Bruno, Claudio ; di Vito, Raffaella ; Vercelli, Alessandro

Spinal Muscular Atrophy (SMA) is a progressive neuromuscular disorder caused by homozygous loss of the survival motor neuron 1 (SMN1) gene, leading to reduced SMN protein expression. Increasing evidence implicates neurotransmission deficits in the pathophysiology of SMA. In particular, alterations in neuroactive amino acids involved in glutamatergic neurotransmission have recently been identified in both the cerebrospinal fluid (CSF) of SMApatients and the spinal cord of SMNΔ7 mouse models. L-arginine, a precursor of nitric oxide, plays a critical role in glutamatergic receptor signalling, influencing neurotransmitter release, synaptic plasticity, and neuroprotection. However, it remains unclear whether SMN deficiency affects L-arginine metabolism in SMA. To address this, we used high-performance liquid chromatography to investigate whether SMN deficiency alters L-arginine homeostasis in the central nervous system of SMNΔ7 mice and in the CSF of SMA patients with varying disease severity, both before and after treatment with the SMN-inducing therapy Nusinersen. Notably, we observed significantly reduced L-arginine levels in the brainstem and spinal cord of symptomatic SMA mice compared to age-matched wild-type littermates. Consistent with these findings, we revealed lower L-arginine levels in severe SMA1 patients compared to milder SMA2 and SMA3 patients and healthy controls, enhancing the translational strength of our findings. Importantly, Nusinersen-mediated SMN upregulation fully restored L-arginine homeostasis in the CSF of severe SMA1 patients. In conclusion, our results demonstrate a dysregulation of L-arginine in SMA and highlight a role for SMN-enhancing therapies in restoring neurochemical alterations observed in patients with this neurodegenerative disease.

743

项与诺西那生钠相关的新闻（医药）

2025-09-02

·BioPharmaDive

Ionis looks to expand use of lipid-lowering drug after trial hits goal

Dive Brief:Ionis on Tuesday said its new lipid-lowering drug Tryngolza met the main goals of two Phase 3 clinical trials that could expand its use. Results showed the drug significantly reduced triglyceride levels in people with severely elevated levels of the fat in their bloods and prevented accompanying pancreatitis episodes.The company will ask the Food and Drug Administration by the end of the year to add severe hypertriglyceridemia to Tryngolzas label. The drug, Ionis first wholly owned product, gained approval in December for a rare inherited condition called familial chylomicronemia syndrome, for which it recorded $25.6 million in sales over the first six months of 2025.Ionis shares rose as much as 34% in morning trading. Leerink Partners analyst Mani Foroohar wrote in a note to clients that the data easily meet our blue-sky scenario for the drug because it gives Tryngolza a bolstered position in a multi-billion dollar market.Dive Insight:Ionis specializes in drugs that block RNA responsible for production of proteins implicated in certain genetically driven diseases. For example, its drug Spinraza, which Biogen sells, treats spinal muscular atrophy, which can be a fatal condition for many babies and children.Until the launch of Tryngolza, Ionis strategy was to license drugs to larger partners like Biogen and AstraZeneca, which have larger commercial arms to reach more physicians and patients. Since Tryngolza, Ionis has also launched a second wholly owned drug called Dawnzera for hereditary angioedema.Tryngolza, which Ionis launched with a list price of $595,000 a year, is approved for a rare condition that affects as many as one in 300,000 people. The potential addition of people with severely elevated triglycerides a condition that is growing with prevalence of obesity and diabetes represents a large expansion opportunity for Tryngolzas market.Called Core and Core2, Ionis trials enrolled 617 and 446 people with severely elevated triglycerides and randomized them to take 80 milligrams or 50 milligrams of Tryngolza every four weeks, or placebo. In Core, the 80 or 50 milligram doses of the drug reduced triglycerides by 73% and 63% respectively, and in Core2 the reductions were 68% and 63%, respectively.In Core, people who received placebo experienced triglyceride lowering of less than 1% on average, while in Core2 it was 14%. The reductions seen in people taking Tryngolza were statistically significant compared with the placebo.Treatment with Tryngolza reduced the incidence of acute pancreatitis by 85% compared with placebo, but Ionis didnt release results from the individual dose arms.These results reinforce our confidence that olezarsen has the potential to change the [severe hypertriglyceridemia] treatment paradigm, said Sam Tsimikas, Ionis senior vice president for global cardiovascular development, in a statement.Jefferies analyst Akash Tewari raised his banks peak sales forecast for Tryngolza from $1.5 billion to $2.5 billion, with the elevated triglyceride indication in the U.S. accounting for about $1.5 billion of the total. '

临床结果上市批准临床3期

2025-08-27

·国家药品监督管理局

2025年08月27日药品批准证明文件送达信息

2022年11月1日起，行政相对人可登录国家药品监督管理局政务服务门户的法人空间查看电子证照，按照相关提示自行打印。序号受理号药品名称申请人批准文号批准日期1CYHB2350964注射用头孢哌酮钠舒巴坦钠广州白云山天心制药股份有限公司————2025年08月25日2CYHB2450217盐酸维拉帕米注射液上海禾丰制药有限公司————2025年08月25日3CYHB2450379维生素B₆注射液河北天成药业股份有限公司国药准字H202581702025年08月25日4CYHB2450416注射用哌拉西林钠他唑巴坦钠山东安信制药有限公司————2025年08月25日5CYHB2450417注射用哌拉西林钠他唑巴坦钠山东安信制药有限公司————2025年08月25日6CYHB2450438氯化钾注射液安徽双鹤药业有限责任公司国药准字H202581682025年08月25日7CYSB2400162卡度尼利单抗注射液康方药业有限公司国药准字S202200182025年08月25日8JYHB2400559曲氟尿苷替匹嘧啶片大鹏药品信息咨询（北京）有限公司————2025年08月22日9JYHB2400560曲氟尿苷替匹嘧啶片大鹏药品信息咨询（北京）有限公司————2025年08月22日10JYHB2400608恩格列净片勃林格殷格翰(中国)投资有限公司————2025年08月25日11JYHB2400609恩格列净片勃林格殷格翰(中国)投资有限公司————2025年08月25日12JYHB2400610恩格列净片勃林格殷格翰(中国)投资有限公司————2025年08月25日13JYHB2400611恩格列净片勃林格殷格翰(中国)投资有限公司————2025年08月25日14JYHB2400612恩格列净片勃林格殷格翰(中国)投资有限公司————2025年08月25日15JYHB2500270来那帕韦钠片吉利德（上海）医药科技有限公司————2025年08月25日16JYHB2500334诺西那生钠注射液渤健生物科技（上海）有限公司————2025年08月25日17JYSB2400079甘精胰岛素注射液礼来苏州制药有限公司————2025年08月22日18JYSB2400080甘精胰岛素注射液礼来苏州制药有限公司————2025年08月22日

申请上市

2025-08-24

·丁香园 Insight 数据库

下一座金矿浮出水面

无论是从技术突破、临床价值，还是商业前景来看，小核酸药物都迎来了发展的黄金时代。今年，受 RNAi 疗法 Amvuttra 销售表现远超预期等因素的带动，Alnylam 股价强势上扬 95%，市值也一举突破了 600 亿美元大关。而 Ionis 和 Avidity 等小核酸上市企业表现同样稳健，2025 年以来股价涨幅分别超过 50% 和 20%。与此同时，凭借靶标选择广泛、治疗效果持久等独特优势，小核酸药物也正在成为投资机构和制药巨头眼中的下一个掘金高地。据不完全统计，今年以来，国内已经有 8 家小核酸企业获得了融资，其中浩博医药、靖因药业、瑞博生物均完成了超 2 亿元融资。海外方面，诺华、礼来、艾伯维、赛诺菲等制药巨头亦通过交易并购不断加码小核酸赛道。来源：Insight 数据库整理（仅统计 IPO 前融资）资本和产业的竞相追逐，无疑佐证了小核酸药物巨大的市场潜力。小核酸药物已经来到商业爆发的前夜。四十年沉浮，终获突破小核酸赛道的发展，经历了长达四十年的技术沉淀和突破。小核酸药物包括反义寡核苷酸（ASO）、小干扰 RNA（siRNA）、微小 RNA（miRNA）、适配体、抗体核酸偶联药物（AOC）等，它们主要通过碱基互补配对原则作用于细胞内的 mRNA，调节基因表达和蛋白质水平，从而发挥治疗作用。早在 1978 年，Zamecnik 和 Stephenson 博士就提出了 ASO 的概念，但学术向产业转化过程总是充满困难的，直到 1998 年，全球首款 ASO 药物福米韦生才获 FDA 批准上市，用于治疗获得性免疫缺陷综合征患者并发的由巨细胞病毒引起的视网膜炎。可惜随着高效抗逆转录病毒疗法的发展，福米韦生市场需求严重萎缩，上市仅 4 年（2002 年）就在欧洲退市。 20 世纪 90 年代以来，RNA 适配体、miRNA、RNAi 先后被发现，进一步掀起了小核酸药物研发的热潮。然而，由于潜在免疫原性、递送系统缺乏等，小核酸药物研发进展始终缓慢，临床研究也屡屡受挫。2010 年，诺华和罗氏终止了与 Alnylam 的合作，随后辉瑞和雅培也放弃了 RNAi 项目的开发。制药巨头的先后离场，使得这个行业陷入停滞。转折发生在 2014 年，Ionis 和 Alnylam 分别在期刊上发表了 N-乙酰半糖胺（GalNAc）-ASO 和 GalNAc-siRNA 偶联技术。这个递送系统的出现，让小核酸药物重新焕发出了生机。 GalNAc 是唾液酸受体（ASGPR）的靶向配体，具有高度肝脏靶向性，通过较低药物剂量便可实现长效，毒性较小。2019 年，首款 GalNAc-siRNA 药物 Givosiran 获 FDA 批准上市。2023 年 12 月，首款GalNAc-ASO 药物 Eplontersen 获 FDA 批准上市。来源：Alnylam 公司官网不过由于 ASGPR 仅在肝实质细胞特异性高表达，GalNAc 递送系统仍存在组织局限性。为拓展到更多疾病领域，小核酸企业还在不断验证新型递送平台，比如Alnylam 开发了能够靶向 CNS、眼部和肺部的 C16 平台等。而得益于化学修饰和递送技术的革新，小核酸药物也开始进入收获期。小核酸为何能成为新宠？ Insight 数据库显示，目前全球在研的小核酸新药已经超过 1200 款，其中获批上市的有 22 款（包含撤市的产品），包括 7 款 siRNA 药物、13 款 ASO 药物和 2 款适配体。来源：Insight 数据库整理从获批上市的产品来看，小核酸已经在罕见病适应症上率先获得突破，包括杜氏肌营养不良、脊髓性肌肉萎缩症、转甲状腺素蛋白淀粉样变性、血友病、家族性高乳糜微粒血症等，与此同时开始逐步拓展至慢性疾病适应症。英克司兰钠（商品名：Leqvio）是全球首款用于降血脂的 siRNA 药物，2021 年 12 月首次在美国获批上市。与传统化药和单抗相比，该产品每半年注射一次便可达到良好的降低 LDL-C 效果。英克司兰钠目前已经在中、美、日、欧等多个国家和地区斩获监管批准，由Alnylam 和诺华共同负责商业化。该产品自上市以来销售额一路突飞猛进，今年第二季度收入 2.98 亿美元，上半年合计收入 5.55 亿美元，有望成为新的重磅炸弹。来源：华福证券小核酸药物在慢性疾病领域的临床和商业价值得到验证，是药企和资本竞相涌入这个赛道的关键原因之一。根据 Insight 数据库，除高血脂外，目前小核酸药物在研适应症已广泛覆盖乙肝、痛风、IgA 肾病、代谢相关脂肪性肝炎（MASH）等疾病，这些均为患者基数庞大、临床需求长期未被充分满足的大适应症，商业前景广阔。作为单抗、小分子后的第三大药物类型，小核酸的技术特性同样赋予了其不可替代的竞争优势。与传统药物相比，小核酸药物不仅研发周期更短，可靶向的分子范围更广，给药后疗效持续时间更长，研发成功率也更高。这些独特优势的叠加，进一步点燃了行业和资本布局小核酸赛道的热情。 Insight 数据库显示，近三年全球一共达成了近百起小核酸 BD 合作，交易金额也在逐年提升。尤其值得关注的是，今年刚刚过半，首付款金额就已经创下新高，这一鲜明的市场信号，正是小核酸药物临床价值与商业价值持续提升的直接印证。来源：Insight 数据库（截至 8 月 21 日）西方亮，东方也要亮与海外相比，我国小核酸药物起步相对较晚，目前仅有 3 款产品上市，还都是进口新药，包括渤健和 Ionis 合作开发的诺西那生钠、托夫生，以及诺华和 Alnylam 合作开发的英克司兰钠。尽管还未有本土产品上市，但我国小核酸赛道已经快速崛起，发展势头强劲。一方面，自 2024 年 1 月以来，瑞博生物、舶望医药、靖因药业等企业先后与海外药企达到了 BD 大单，合作对象不乏勃林格殷格翰、诺华等 MNC，交易金额最多的高达 50 亿美元。另一方面，国产小核酸新药研发管线正在快速扩充，数量仅次于美国。目前，在研新药已经达到了 300 多款，进入临床阶段的也有 68 款。从技术类型来看，国内小核酸临床管线呈现出明显的单极主导特征——siRNA 疗法占据绝对主流，而 ASO、小激活 RNA、miRNA 等疗法仍较少，在研新药项目仅有个位数。来源：Insight 数据库在适应症的选择上，与海外率先突破罕见病不同，国内大部分小核酸企业直接将视线瞄向了心血管代谢、肝病、痛风等慢性疾病领域。比如国产进展最快的AHB-137 针对的适应症就是慢性乙肝。来源：Insight 数据库 AHB-137 是浩博医药基于 Med-Oligo™ 平台开发的一款非偶联型 ASO 药物，正在开展慢性乙肝的 III 期临床。今年 5 月，浩博医药在欧洲肝病学会年会上首次披露了该产品 IIb 期临床的中期数据。这项研究一共纳入了 64 名受试者，随机分组接受AHB-137 300 mg 24 周治疗和 16 周治疗。结果显示，在24周治疗组中，75% 受试者达到了主要终点，乙型肝炎表面抗原（HBsAg）水平低于定量下限（<0.05 IU/mL）以及乙型肝炎病毒（HBV）DNA 水平低于定量下限（<10 IU/mL）。在 16 周治疗组中，66% 受试者达到了这一主要终点。在达到主要终点的受试者中，超过 80% 的受试者在治疗 12 周内实现了 HBsAg 的清除，并且在治疗结束时，24 周和 16 周组分别有 54% 和 33% 的受试者出现了乙肝表面抗体（anti-HBs）的血清转换。AHB-137 在两个治疗组中均表现出良好的耐受性和有效的安全性。来源：Insight 数据库面对乙肝这一庞大的市场，舶望制药、恒瑞医药、瑞博生物、正大天晴等国内药企都布局了小核酸药物。不过与 AHB-137 不同，它们开发的都是 GalNAc-siRNA 疗法，目前尚处于早期临床阶段（II 期和 I 期）。事实上，放眼全球，在乙肝、高脂血症、高血压等慢性疾病领域的小核酸药物研发赛道上，我国都已展现出强大的竞争力。国内药企通过密集的管线布局和高效的临床推进，已经建立起全球前列的研发梯队。这种战略聚焦不仅贴合国内医疗需求的实际痛点，更在全球小核酸药物的应用版图中开辟出了差异化竞争赛道。结语在竞争激烈的创新药环境中，每个参与者都渴望成为第一个撞线的人。当 ADC 与 GLP-1 的热潮逐渐理性，小核酸正成为制药巨头与投资人眼中的下一座金矿。封面来源：站酷海洛免责声明：本文仅作信息分享，不代表 Insight 立场和观点，也不作治疗方案推荐和介绍。如有需求，请咨询和联系正规医疗机构。编辑：月牙 PR 稿对接：微信 insightxb 投稿：微信 insightxb；邮箱 insight@dxy.cn

上市批准siRNA寡核苷酸并购核酸药物

100 项与诺西那生钠相关的药物交易

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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,

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适应症	国家/地区	公司	日期
神经肌肉疾病	中国	Biogen Netherlands BV	2019-02-22
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,

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	西班牙	Biogen, Inc.	2014-08-19
I型脊髓性肌萎缩症	临床3期	瑞典	Biogen, Inc.	2014-08-19

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临床结果

适应症

分期

评价

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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


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))	獵餘衊網鏇襯鑰顧繭獵(襯網顧蓋餘憲選鹽獵餘) = 鏇顧蓋獵構築廠淵憲憲鬱範壓鹽繭選糧鹹齋獵 (範淵築蓋艱夢選製蓋繭, 0.49 ~ 4.56) 更多	积极	2025-06-27
NCT02386553 (NURTURE, Company_Website) 人工标引	临床2期	脊髓性肌萎缩	25	SPINRAZA 12mg	網鏇夢築繭艱窪製餘衊(餘壓廠襯壓觸艱繭醖壓) = 網醖簾淵獵窪糧鹽觸齋鹽觸壓壓餘艱壓鏇衊鏇 (鏇簾糧餘鏇壓製範淵構 ) 更多	积极	2025-06-27
NCT02594124 (SHINE Study (1692) \| SHINE, CTgov)	临床3期	脊髓性肌萎缩	292	nusinersen (Infantile SMA Onset CS3A)	鏇鏇遞簾鏇鬱夢顧築鏇 = 鑰壓鏇廠蓋鏇蓋蓋衊觸鏇範顧蓋鏇艱積構範襯 (襯齋遞積糧鑰構淵衊艱, 鏇憲襯觸獵餘壓製窪鹽 ~ 繭願醖艱夢觸選鬱鑰蓋) 更多	-	2024-10-22
NCT02594124 (SHINE Study (1692) \| SHINE, CTgov)	临床3期	脊髓性肌萎缩	292	nusinersen (Infantile SMA Onset CS3B Previous Control)	鏇鏇遞簾鏇鬱夢顧築鏇 = 築夢獵網願鹽糧夢糧齋鏇範顧蓋鏇艱積構範襯 (襯齋遞積糧鑰構淵衊艱, 繭窪衊鏇鑰糧壓願願糧 ~ 膚願膚襯構鹽鑰築選憲) 更多	-	2024-10-22
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	醖選襯遞鹽願鬱遞艱艱(繭壓憲蓋淵繭衊鏇壓觸) = 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. 選築齋衊夢齋餘構鹽窪 (積範糧艱築選醖構鬱顧 ) 达到更多	积极	2024-09-04
NCT04089566 (DEVOTE, NEWS) 人工标引	临床2/3期	脊髓性肌萎缩	75	Spinraza 28/28 Milligram (mg) Safety Group		积极	2024-09-04
NCT04419233 (PANDA, Pubmed \| Adv Ther)	临床4期	脊髓性肌萎缩	50	Nusinersen	積範艱獵顧鬱醖夢壓鹹(鹽齋鹽範構糧衊夢選鹽) = 夢鹽廠鑰醖夢淵築獵網積夢餘艱糧簾鏇獵艱廠 (鑰遞製願壓糧鑰繭鏇築 )	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	蓋齋艱壓築鹹築觸簾夢(襯鹽艱繭鬱顧壓襯範襯) = 鹽積鹽遞製壓鏇遞顧觸窪齋醖廠鹹積鏇築糧襯 (築憲窪蓋餘築鏇顧艱膚 ) 更多	不佳	2024-03-06
NCT04488133 (RESPOND, AAN2023) 人工标引	临床4期	脊髓性肌萎缩二线 SMN2 copy	34	Nusinersen 12mg	範鹽獵觸簾糧範網餘築(鬱鹽糧鏇鹹窪網蓋製鬱) = Upper respiratory tract infection (n=6) and viral upper respiratory tract infection (n=5) 鹹壓願觸鏇鹽鑰鬱鏇願 (獵艱範壓餘鏇築齋鹹顧 )	不佳	2023-03-19
NCT02386553 (NURTURE, MDA2023) 人工标引	临床2期	脊髓性肌萎缩 SMN2 copies	15	Nusinersen (2 SMN2 copies)	簾鹹構鑰構齋窪衊範鏇(鹹憲製鹽糧艱襯夢糧艱) = 膚蓋襯鏇範壓簾鹹獵糧夢膚壓鏇構範鬱糧壓蓋 (遞積窪鏇夢襯鬱鹽憲鏇 ) 更多	积极	2023-03-19
NCT02386553 (NURTURE, MDA2023) 人工标引	临床2期	脊髓性肌萎缩 SMN2 copies	15	Nusinersen ( 2 SMN2 copies who did not have CMAP <2 mV )	簾鹹構鑰構齋窪衊範鏇(鹹憲製鹽糧艱襯夢糧艱) = 鹽襯糧醖憲憲顧憲築壓夢膚壓鏇構範鬱糧壓蓋 (遞積窪鏇夢襯鬱鹽憲鏇 ) 更多	积极	2023-03-19
NCT04488133 (RESPOND, MDA2023)	临床4期	脊髓性肌萎缩 SMN2 copies	34	Nusinersen	製願積範襯遞觸醖簾網(觸選繭鹽鑰繭顧鏇鏇鏇) = No deaths occurred 餘築餘窪襯糧顧廠淵糧 (鏇鏇夢廠遞窪鏇壓壓壓 ) 更多	积极	2023-03-19
Pubmed 人工标引	N/A	脊髓性肌萎缩	401	Nusinersen sodium (infantile-onset SMA)	顧鏇艱製餘襯簾蓋醖構(膚簾簾積糧憲選簾壓鬱) = 範夢顧簾築鏇觸齋餘鑰憲齋艱餘製鬱餘壓製衊 (夢廠網製鏇淵製鹹糧觸 )	积极	2022-07-05
Pubmed 人工标引	N/A	脊髓性肌萎缩	401	Nusinersen sodium (later-onset SMA)	顧鏇艱製餘襯簾蓋醖構(膚簾簾積糧憲選簾壓鬱) = 範範蓋積鬱齋願願製鑰憲齋艱餘製鬱餘壓製衊 (夢廠網製鏇淵製鹹糧觸 )	积极	2022-07-05