Onasemnogene Abeparvovec: ASsessing The REAl-world Safety & Effectiveness of Spinal Muscular Atrophy Participants Treated With Intrathecal Onasemnogene Abeparvovec-brve (ITVISMA®): A U.S. Pragmatic Multicenter Study (STREAM)

The primary purpose is to address critical evidence in the treatment landscape for Spinal Muscular Atrophy (SMA), specifically focusing on the intrathecal formulation of onasemnogene abeparvovec-brve (ITVISMA®). U.S. Pragmatic Multicenter Study (STREAM).

开始日期2026-08-27

申办/合作机构

Novartis Pharmaceuticals Australia Pty Ltd.

NCT06019637

/ RecruitingN/A

A Long-term Safety Study in Brazilian Patients With a Confirmed Diagnosis of Spinal Muscular Atrophy (SMA) Treated With Onasemnogene Abeparvovec (Zolgensma®) - ARISER Study

A long-term safety study in Brazilian patients with a confirmed diagnosis of Spinal Muscular Atrophy (SMA) treated with Onasemnogene Abeparvovec (Zolgensma®)

开始日期2023-11-27

申办/合作机构

Novartis Pharmaceuticals Corp.

IRCT20221024056284N1

/ CompletedN/AIIT

Evaluation the color match of two single shade resin composites; Charisma Diamond ONE and Charisma Topaz ONE in class five restorations and assessment of dentist and patient satisfaction

开始日期2023-01-21

申办/合作机构

Mashhad University of Medical Sciences

100 项与阿哌奥诺基相关的临床结果

登录后查看更多信息

100 项与阿哌奥诺基相关的转化医学

登录后查看更多信息

100 项与阿哌奥诺基相关的专利（医药）

登录后查看更多信息

369

项与阿哌奥诺基相关的文献（医药）

2027-03-01·Value in Health Regional Issues

Cost-Effectiveness of Universal Newborn Screening and Disease-Modifying Therapies for Spinal Muscular Atrophy in Taiwan

Article

作者: Hsieh, Hui-Min ; Chen, Huey-Fen ; Prosser, Lisa A ; Jong, Yuh-Jyh ; Chiang, Wan-Ling ; Chen, Chung-Yu

OBJECTIVES:

Spinal muscular atrophy (SMA) is a rare neuromuscular disorder and an important genetic cause of infant mortality. Universal newborn screening (NBS) enables presymptomatic disease-modifying therapy (DMT) but increases upfront costs. This study evaluated the cost-effectiveness of universal SMA NBS in Taiwan.

METHODS:

This study built a state-transition simulation model for a hypothetical SMA birth cohort using local epidemiology and costs and published clinical inputs. Strategies were as follows: (1) NBS with presymptomatic DMTs and (2) clinical identification (CI) with symptomatic DMTs. DMTs included nusinersen, onasemnogene abeparvovec, and risdiplam (reimbursed by Taiwan's National Health Insurance). Incremental costs, quality-adjusted life-years (QALYs), and incremental cost-effectiveness ratios (ICERs) were estimated over 10-year and lifetime (90-year) horizons from Taiwan's payer and societal perspectives. The willingness-to-pay (WTP) threshold was 3 times Taiwan's 2022 GDP per capita (New Taiwan Dollar (NTD) 2 917 650/QALY). One-way, probabilistic sensitivity, and scenario analyses assessed uncertainty.

RESULTS:

All ICERs were below WTP threshold. From the payer perspective, ICERs for NBS with presymptomatic DMT versus CI with symptomatic DMT were NTD 1 157 970 (USD 38 599) /QALY (10 years) and NTD 2 108 499 (USD 70 283)/QALY (lifetime). From the societal perspective, ICERs were NTD 261 789 (USD 8726)/QALY (10 years) and NTD 1 494 002 (USD 49 800)/QALY (lifetime). Results were most sensitive to DMT costs (particularly nusinersen and risdiplam) and health-state utility values.

CONCLUSIONS:

Universal SMA NBS with presymptomatic DMT is likely cost-effective in Taiwan from both payer and societal perspectives under the WTP threshold, supporting earlier treatment to slow progression and improve quality of life.

2026-11-01·ADVANCES IN THERAPY

Longitudinal Dosing Patterns and Treatment Costs Among Patients with Spinal Muscular Atrophy Initiating Nusinersen and Risdiplam

Article

作者: Song, Wei ; Ye, Dongni ; Yang, Min ; Toro Jimenez, Walter ; Lemus Wirtz, Esteban

INTRODUCTION:

Spinal muscular atrophy (SMA) is a neuromuscular disorder historically associated with high morbidity, mortality, and healthcare costs. Disease-modifying therapies have improved outcomes, but their long-term economic burden remains uncertain. Nusinersen and risdiplam, both chronic therapies, differ from onasemnogene abeparvovec, a one-time gene therapy. Understanding real-world treatment costs of chronic SMA therapies is critical to inform therapeutic choice.

METHODS:

This retrospective cohort study used the Komodo Health Research Database (2016-2024). Patients with SMA aged ≥ 2 years who initiated nusinersen or risdiplam were followed up to 5 years. Dosing patterns and drug acquisition and administration costs (2024 USD) were assessed annually, overall and by age group and scoliosis status.

RESULTS:

Among 3864 eligible patients, 2743 initiated nusinersen and 1121 initiated risdiplam (mean age 21.4 vs. 25.3 years, respectively; 51.5% vs. 46.1% male). Median follow-up was 5.5 years for nusinersen and 1.1 years for risdiplam. Persistence declined over time, with 39.5% of patients treated with nusinersen and 63.7% of patients treated with risdiplam remaining on their index therapy at years 5 and 4. Patients treated with nusinersen received 3.6 injections on average in year 1 and 2.8-3.1 annually thereafter. Median annualized nusinersen treatment costs were $504,173 in year 1 and $438,788-548,608 in years 2-5. Patients treated with risdiplam had a median of 96 days of supply in year 1 (51.5% with > 90 days); median annualized drug costs were $143,331 overall in year 1 and $259,419 among those with > 90 days of supply, increasing to $264,701-289,340 in years 2-4. Costs were similar across subgroups.

CONCLUSION:

Nusinersen and risdiplam persistence declined over time. Among patients remaining on their index therapy, both treatments had high long-term costs, highlighting the substantial and ongoing financial burden of chronic SMA therapies and the need for real-world evidence to inform healthcare planning.

TRIAL REGISTRATION:

ClinicalTrials.gov identifier, NCT07378943.

2026-11-01·Journal of Neuromuscular Diseases

Determinants of hepatic enzyme elevations following onasemnogene abeparvovec: Results from a unified immunomodulation protocol

Article

作者: Dhawan, Anil ; Parashar, Syon ; Mundada, Vivek

Introduction:

Onasemnogene abeparvovec is an adeno-associated virus serotype 9 (AAV9) gene therapy for spinal muscular atrophy (SMA) that is frequently associated with immune-mediated hepatotoxicity. Previous studies have suggested that older age and higher body weight may increase the risk or severity of hepatic enzyme elevation; however, interpretation is limited by heterogeneous immunosuppression strategies across centers.

Method:

We conducted a retrospective cohort study of children with SMA who received onasemnogene abeparvovec between 2020 and 2025 and were managed using a unified, protocol-driven immunomodulation strategy. Peak alanine aminotransferase (ALT) and aspartate aminotransferase (AST) values (upper limit of normal [ULN] 35 U/L), as well as the timing of peak transaminase elevations, were analyzed in relation to age and weight at infusion.

Results:

Among 152 children with adequate biochemical follow-up, hepatic enzyme elevation was common: 63.2% had peak ALT ≥2× ULN and 29.6% had peak ALT ≥5× ULN. Weight demonstrated a statistically significant but modest correlation with peak ALT magnitude (r = 0.184, p = 0.023), whereas age was not associated with transaminase severity. In contrast, age correlated with delayed timing of peak ALT (r = 0.229, p = 0.005) and AST (r = 0.283, p < 0.001). All hepatic enzyme abnormalities resolved under protocol-based immunomodulation, and no cases of hepatic failure or synthetic dysfunction occurred.

Conclusion:

When managed with a structured, proactive immunomodulation protocol, hepatotoxicity following onasemnogene abeparvovec is predictable and manageable. Weight modestly influences ALT severity, while age primarily affects the timing rather than the magnitude of hepatic immune activation, supporting safe administration across a broad pediatric age and weight spectrum.

1,661

项与阿哌奥诺基相关的新闻（医药）

2026-11-11

·医药代表

突发，诺华被调查！！

据罗湿兄报道，近日，FDA开启专项调查，直指诺华两款SMA基因疗法存在致癌风险，其中就包括曾登顶全球最贵药物的Zolgensma。这款千万级天价救命药，一度是脊髓性肌萎缩症患儿的治愈希望，如今深陷安全争议，引发整个生物医药圈热议。Zolgensma在2019年获批上市，专门治疗2岁以下的SMA患儿，依靠单次给药根治疾病的优势，迅速成为重磅特效药。其上市定价高达212.5万美元，折合人民币超1400万，常年稳居全球最贵药物榜单。凭借独一无二的治疗优势，该药商业成绩亮眼，全球已有超6000名患者使用，累计销售额突破76亿美元，即便今年依旧保持强劲营收。此次风险争议的核心，是基因疗法的插入性致癌隐患。这款药物依靠AAV病毒载体，将正常基因送入人体修复病灶。但病毒载体存在极低概率整合进人体基因组，一旦嵌入癌基因区域，就可能激活致癌突变，这也是基因疗法行业一直存在的理论风险。本次FDA调查有据可依，目前全球已收到9例患者用药后罹患肿瘤的报告。最关键的是，其中2例患者的检测结果实锤风险：治疗用的AAV载体，精准整合到了人体GLI1癌基因附近，直接诱发肿瘤生长。这意味着曾经的理论风险，已经变成真实发生的临床安全事故。值得注意的是，同技术路径的诺华新药Itvisma，因使用同款病毒载体，也被一并纳入调查。诺华方面紧急回应，称仅2例可证实药物关联，其余病例暂未发现因果关系，且相关风险早已标注在药品说明书中。目前FDA并未叫停药物使用，明确现阶段药物获益依旧大于潜在风险，但会持续评估后续数据，不排除出台新的监管限制。天价、特效、一次性治愈，是Zolgensma的标签，也是基因疗法行业的天花板。此次致癌风波，彻底撕开了高端基因疗法的隐藏短板。看似完美的前沿医疗技术，依旧存在难以规避的未知风险，也为整个基因治疗赛道的研发和监管，敲响了一记响亮的警钟。免责声明：本文仅为行业资讯客观梳理，不构成任何诊疗建议、投资建议，药品最终使用请以官方审批说明及医师指导为准。

基因疗法上市批准

2026-11-11

·BioSpace

Novartis’ Zolgensma woes, BIO’s China plan, GLP-1s’ new safety warning, FDA’s stacked November

Novartis confirms that nine patients treated with its spinal muscular atrophy gene therapy Zolgensma have developed tumors; the FDA orders GLP-1 drugmakers to add a warning to their labels about the risk of a rare eye condition that can lead to vision loss; and the agency is set to decide on no fewer than 11 therapies this month, including closely watched applications from Summit Therapeutics, Vertex and Capricor Therapeutics. > Listen on Spotify > Listen on Apple Products > Listen on Amazon Music > Listen on iHeart > Watch on YouTube This biggest news of the past week was undoubtedly the revelation of tumors developing in patients with spinal muscular atrophy who received Novartis’ Zolgensma. The FDA has now launched an investigation into the approved gene therapy. Then, to add insult to injury, the Swiss pharma revealed detailed results from its failed HORIZON cardiovascular trial , and no matter which way the data were cut, the results remained negative. Novartis and partner Ionis revealed in September that pelacarsen, which lowers levels of lipoprotein(a), failed to show efficacy in lowering the risk of major adverse cardiovascular events. With the new data offering “little silver lining,” according to Truist Securities, Lp(a) as a target in cardiovascular disease is being further scrutinized. On a global scale, China has been on the rise when it comes to biotech, as evidenced by the volume of licensing deals going to Chinese companies. The Asian country has been working toward this goal for a long time, with the government releasing carefully curated strategic plans every five years for the past few decades. Now, the Biotechnology Innovation Organization (BIO) has released a proposal laying out seven policies the U.S. government can work on to help protect the American biotech industry. In the weight loss space, the FDA is requiring a label update for all 12 GLP-1 receptor agonists currently on the market to now include a potential increased risk for a rare eye disease that causes optic nerve swelling and can lead to blindness. Cases of the disease have been reported for all currently marketed GLP-1s, according to the FDA, including Novo’s semaglutide drugs Wegovy and Ozempic and Eli Lilly’s tirzepatide franchise, Zepbound and Mounjaro, as well as its oral GLP-1, Foundayo. On the oral front, frontrunners Novo and Lilly have been duking it out for market share. In its third quarter earnings call last week, Novo said Lilly’s claim that Foundayo is closing on in the Wegovy pill isn’t quite right. Meanwhile, Lilly released results from a Phase 3 sleep apnea trial of Foundayo—upon which the company will seek its second approval in the indication; Lilly’s first sleep apnea approval—and the first overall for the condition—was for its injectable Zepbound in 2024. Meanwhile, Zealand and Boehringer Ingelheim are one step closer to global regulatory filings for their dual glucagon and GLP-1 receptor agonist survodutide after a late-stage trial showed the drug did not raise the risk of cardiovascular death. The FDA has a full docket this month, including pending decisions for Summit Therapeutics and Akeso’s bispecific antibody ivonescimab, Capricor’s Duchenne muscular dystrophy cell therapy deramiocel and Vertex’s povetacicept in IgA nephropathy. Finally, check out BioSpace ’s 2027 Best Places to Work list , which includes Vertex and 49 other life sciences employers.

临床3期上市批准临床结果细胞疗法基因疗法

2026-11-10

·云健康生活

今日简讯：创新药进院考核松绑、流感原料药获批、FDA确认一款基因疗法致癌信号

1. 四部门发文：创新药进医院，松了一道关键的绑国家卫健委、医保局、中医药局、疾控局联合印发通知，核心一条：创新药不纳入门诊次均药品费用增幅、住院次均药品费用增幅等公立医院绩效监测指标，同时不对医院总药品品种品规数量做限制。对普通人意味着什么：过去医院控费压力大，贵的创新药进院难、医生开起来有顾虑。这道“考核枷锁”松动后，已经获批的新药进医院的速度有望加快，患者在院内用到前沿疗法的可及性会改善。但落地节奏取决于各医院药事会的执行。 2. 流感药“原料端”多了一个玩家，但别急着等降价珍宝岛的玛巴洛沙韦原料药获国家药监局批准注册，适用于成人和儿童流感治疗。关键区分：这次批的是原料药，不是制剂。原料药获批意味着后续仿制药生产有了原料基础，但离患者买到便宜的玛巴洛沙韦制剂还有距离。流感季临近，这条的长期意义大于短期。 3. 慢性荨麻疹：一款口服新药进入临床，国内同靶点尚无上市药 ST人福的HW281171片获批临床试验，化学药品1类，口服小分子，拟用于慢性荨麻疹。经数据库统计，国内目前暂无同靶点同适应症药物上市，已有2家公司进入I期临床。对患者的含义：抗组胺药控制不佳的慢性荨麻疹患者，未来可能多一个口服选择。但刚进临床，距离上市通常还有数年。 4. 司美格鲁肽的“肾脏保护”专利延长到2031年诺和诺德诺和泰（司美格鲁肽注射液）慢性肾脏病适应症的专利权期限补偿获批，相关专利保护期延长5年至2031年3月。对普通人的双面影响：正面是鼓励企业继续深耕CKD这一适应症；另一面是仿制药或生物类似药进入这一特定适应症的时间窗口被推迟。用得起用不起，最终还是看医保谈判和市场竞争，专利延期不等于价格一定居高不下，但短期内降价压力会小一些。 5. 恒瑞一款“双抗”瞄准掌跖脓疱病，国内外均无同类上市 SHR-1139注射液获批临床试验，同时靶向IL-23p19和IL-36R，用于掌跖脓疱病，目前国内外尚无同类药物获批上市，累计研发投入约1.63亿元。为什么值得关注：掌跖脓疱病是一种反复发作、治疗选择有限的慢性皮肤病，现有疗法多为超说明书使用。如果这条路径走通，是真正意义上的“从0到1”。 6. 甘李药业入局特应性皮炎，口服新药完成首例给药 GLR2053片Ⅰ期临床完成首例受试者给药，拟用于特应性皮炎。甘李以胰岛素闻名，这是向自免/皮肤领域延伸的信号。对AD患者来说，口服小分子如果能做出疗效和安全性，比生物制剂皮下注射更方便。 7. 热景生物心梗/心衰方向创新药Ib期：安全性过关 SGC001在美国心脏协会年会上报告Ib期数据，低中高三个剂量组均未发生3级及以上不良事件，无严重不良事件，无因不良事件停药。心血管领域的新靶点药物能通过早期安全性考验，才有资格进入更大规模的疗效验证。现在谈“有效”还太早。 8. FDA确认一款基因疗法的致癌信号，但别误读到新冠疫苗上 FDA确认诺华Zolgensma（脊髓性肌萎缩症AAV9基因疗法）存在产品相关的插入性致癌：超过6000例治疗患者中报告9例肿瘤，其中2例显示载体DNA整合在GLI1致癌基因附近或内部，第3例仍在调查。需要划清的界限：这是AAV基因治疗的安全信号，有明确的分子机制证据。但部分评论将其引申为“腺病毒新冠疫苗同样致癌”，目前没有证据支持这一跳跃——AAV和腺病毒载体的整合行为、剂量、分布都不同。对患者而言，Zolgensma的获益-风险仍需个体化评估，监管和长期随访会收紧；对公众而言，不应把一种基因疗法的风险泛化到所有疫苗。 9. 全球首个志贺菌疫苗候选，II期双试验同时阳性法国Valneva的四价志贺菌疫苗S4V2，在肯尼亚婴儿中显示良好安全性和四型抗体持续6个月以上；在美国成人受控人类感染模型中，疫苗组志贺症发生率较安慰剂显著下降，达到主要终点。FDA此前已授予快速通道。公共卫生意义：志贺菌是全球腹泻致病和致死的重要原因，目前没有任何获批疫苗。如果后续III期验证，这是真正填补空白的品类。 10. 华纳药厂糖尿病复方仿制药获批，视同过一致性评价二甲双胍恩格列净片（Ⅰ）获药品注册证书，用于正在接受恩格列净和二甲双胍治疗的2型糖尿病成人患者改善血糖控制，按化学药品4类获批，视同通过一致性评价。复方制剂能减少服药片数，对需要联合用药的糖尿病患者是依从性上的改善；仿制药入场也有助于拉低整体费用。 11. 百奥泰花5000万“买回”贝伐珠单抗，剑指生物类似药集采百奥泰从百济神州收回普贝希（贝伐珠单抗）中国全部权益，对价5000万元，前期已收的3.62亿元不退回。背景是贝伐珠单抗已被纳入单抗类生物制剂集采筹备范围，赛道内符合条件的企业达13家。对患者的含义：贝伐珠单抗是多种实体瘤的基础用药。企业争夺自主权，说明价格战在即。集采落地后，患者自付部分有望明显下降，但具体降幅要看中选规则和医保衔接。 12. 创新药审评“30日通道”跑通，但能跑多快看执行国家药监局药品审评中心在虹桥论坛透露，创新药临床试验审评审批试点已跑通“30日通道”。审评提速对患者的意义是新药更早进入可及阶段，但审评快不等于上市快，后续临床试验、注册审批、医保准入每一环都还在。今日科普话题：体检报告上的“窦性心律不齐”，到底要不要紧？很多人拿到心电图报告，看到这几个字就慌了。绝大多数情况下，不用治。 “窦性”说明心律的起搏点仍然是心脏正常的司令部——窦房结，这是对的。 “不齐”指的是心跳间隔有轻微长短变化。健康人的心率本来就不是节拍器，吸气时稍快、呼气时稍慢，叫“呼吸性窦性心律不齐”，在年轻人里非常常见，是迷走神经正常调节的表现。需要认真对待的，是伴随其他描述的情况：比如“窦性心律不齐”同时出现明显的心慌、头晕、黑矇，或者心电图还报了频发早搏、传导阻滞、ST-T改变。单纯一条“窦性心律不齐”、没有任何症状，通常不需要用药，也不需要反复复查。因此，看报告别只看“不齐”两个字，看有没有症状、有没有合并其他异常。拿不准就把完整心电图给心内科医生看一眼，比自己搜半天踏实。 —END— 长期招募合作伙伴，给患者提供更好的服务和赞助支持！如果您有意向可以联系助手，我们将给您定制您的爱心赞助计划！国家药监局申报三甲医院免费临床试验，肿瘤、皮肤病、慢性病、罕见病、医美项目、抑郁症等项目公益咨询、病友群请联系我们。关注我们，服务您更好的生活

上市批准生物类似药基因疗法专利到期

100 项与阿哌奥诺基相关的药物交易

登录后查看更多信息

外链

KEGG	Wiki	ATC	Drug Bank
D11559	阿哌奥诺基	M09AX M09AX09	DB15528

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
I型脊髓性肌萎缩症	韩国	Novartis Gene Therapies, Inc.	2021-05-28
脊髓性肌萎缩	美国	Novartis Gene Therapies, Inc.	2019-05-24

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
青少年脊髓性肌萎缩症	临床3期	美国	Novartis Gene Therapies, Inc.	2020-02-10
青少年脊髓性肌萎缩症	临床3期	日本	Novartis Gene Therapies, Inc.	2020-02-10
青少年脊髓性肌萎缩症	临床3期	澳大利亚	Novartis Gene Therapies, Inc.	2020-02-10
青少年脊髓性肌萎缩症	临床3期	比利时	Novartis Gene Therapies, Inc.	2020-02-10
青少年脊髓性肌萎缩症	临床3期	加拿大	Novartis Gene Therapies, Inc.	2020-02-10
青少年脊髓性肌萎缩症	临床3期	法国	Novartis Gene Therapies, Inc.	2020-02-10
青少年脊髓性肌萎缩症	临床3期	意大利	Novartis Gene Therapies, Inc.	2020-02-10
青少年脊髓性肌萎缩症	临床3期	中国台湾	Novartis Gene Therapies, Inc.	2020-02-10
青少年脊髓性肌萎缩症	临床3期	英国	Novartis Gene Therapies, Inc.	2020-02-10
II型脊髓性肌萎缩	临床3期	日本	Novartis Gene Therapies, Inc.	2018-04-02

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


NCT04488133 (RESPOND, CTgov)	临床4期	脊髓性肌萎缩	46	nusinersen (Nusinersen 2 SMN2 Copies)	齋遞衊簾觸襯憲艱遞鬱(窪齋衊觸願製醖壓憲繭) = 憲壓廠鏇觸憲願窪憲蓋製壓獵顧鬱夢窪餘鑰選 (觸衊鹹繭構壓選簾鹹獵, 4.05) 更多	-	2026-10-30
NCT04488133 (RESPOND, CTgov)	临床4期	脊髓性肌萎缩	46	nusinersen (Nusinersen 3 SMN2 Copies)	齋遞衊簾觸襯憲艱遞鬱(窪齋衊觸願製醖壓憲繭) = 網膚願鹽淵網壓鏇鹹構製壓獵顧鬱夢窪餘鑰選 (觸衊鹹繭構壓選簾鹹獵, 7.89) 更多	-	2026-10-30
NCT04174157 (RESTORE, Pubmed \| Ann Clin Transl Neurol)	N/A	脊髓性肌萎缩	80	Onasemnogene Abeparvovec (Spinal Muscular Atrophy)	選範糧製築築窪構鬱襯(窪鏇餘願衊衊壓製膚範) = 範鹽淵製繭積願窪窪憲鹹膚構壓壓糧醖願齋繭 (製觸餘淵簾憲醖構築繭 ) 更多	积极	2026-06-09
AAN2026	N/A	脊髓性肌萎缩	565	Onasemnogene Abeparvovec Gene Therapy	膚遞鹽網衊窪膚齋壓夢(顧範範餘夢積製廠顧襯) = 選選糧遞壓網淵壓繭範壓鹽鏇鬱鹹醖齋齋壓鏇 (鏇獵顧築襯憲蓋廠鬱製, 12.07 ~ 19.47) 更多	积极	2026-04-25
NCT02122952 \| NCT03421977 (LT-001, Pubmed \| EClinicalMedicine)	临床1期	脊髓性肌萎缩	13	Onasemnogene abeparvovec	齋顧鬱齋夢觸淵築膚餘(積選糧鹹膚鏇網鑰糧範) = 夢鏇鹽醖鹽艱遞夢餘獵齋憲鏇鏇壓觸蓋鹹築衊 (積鑰鏇糧壓憲願蓋鬱壓 ) 更多	积极	2026-04-01
RESTORE Registry (MDA2026)	N/A	脊髓性肌萎缩	251	Onasemnogene Abeparvovec	顧簾襯繭繭顧醖鬱範淵(觸範觸窪簾鑰蓋餘憲網) = 積淵艱範醖願膚獵蓋襯艱艱衊製糧願憲蓋選築 (窪廠壓積廠窪網衊願選 ) 更多	积极	2026-03-08
NCT05089656 (STEER, MDA2026)	临床3期	脊髓性肌萎缩	445	OAV101 IT	築築膚衊鹹鹹繭夢壓齋(築襯壓壓鏇獵選醖襯簾): MD = 1.93 (95.0% CI, -0.25 ~ 4.1) 更多	积极	2026-03-08
NCT05089656 (STEER, MDA2026)	临床3期	脊髓性肌萎缩	445	Risdiplam		积极	2026-03-08
MDA2026	N/A	脊髓性肌萎缩	23	Nusinersen	憲製繭憲壓壓鹽壓觸遞(餘廠積觸積餘鑰鹹製齋) = 願選顧鹽簾觸鏇製窪製餘網繭鏇襯鹹糧衊憲構 (積願窪襯齋衊淵齋糧衊, 1.75 ~ 6.15)	积极	2026-03-08
NCT05089656 (STEER, MDA2026)	临床3期	脊髓性肌萎缩	126	OAV101 IT	鑰獵衊鏇鹹鬱艱顧範衊(簾觸簾構顧鏇築構繭鬱) = 遞鹹鏇餘構繭鏇鏇鏇鑰網繭鏇廠夢憲鏇膚蓋艱 (夢築網鑰顧窪觸鑰範淵, 1.54 ~ 3.27) 更多	积极	2026-03-08
NCT05386680 (STRENGTH, Pubmed \| Nat Med)	临床3期	脊髓性肌萎缩	27	Onasemnogene abeparvovec	醖鹽範遞廠窪醖艱醖鹽(鹹築衊鏇鏇糧積窪鏇淵) = 鬱遞簾築築願築糧壓繭繭憲遞繭鏇餘簾簾膚衊 (簾衊艱選艱選淵窪襯憲 ) 更多	积极	2025-12-08
STRENGTH (WCN2025)	临床3期	脊髓性肌萎缩	59	Onasemnogene abeparvovec	壓壓網鹽構夢蓋鹹遞鑰(蓋夢遞顧壓衊壓膚範範) = The most common AEs were fever (75%, primarily low-grade) and vomiting (47%). Most AEs were mild to moderate in severity and temporary. 積齋鹹選積餘製鏇鹹夢 (鬱蓋夢襯鏇選簾繭淵壓 )	积极	2025-10-12