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更新于：2025-01-08
Nusinersen sodium
诺西那生钠
更新于：2025-01-08
概要
基本信息
非在研机构- |
最高研发阶段批准上市 |
首次获批日期 美国 (2016-12-23), |
最高研发阶段(中国)批准上市 |
特殊审评快速通道 (美国)、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) |
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结构/序列
Sequence Code 29854605
来源: *****
关联
26
项与 诺西那生钠 相关的临床试验NCT06555419
An Open Label, Single Cohort Study to Assess the Pharmacokinetic Profile of Nusinersen (BIIB058) Administered Via the ThecaFlex DRx™ System (PIERREPK)
CTR20232342
一项在既往参加过诺西那生钠试验性研究的脊髓性肌萎缩症受试者中给予高剂量诺西那生钠 (BIIB058) 的长期扩展研究
ChiCTR2200064655
Ultrasound-guided nusinersen administration for spinal muscular atrophy patients with scoliosis: median transverse positioning approach versus paramedian sagittal positioning approach
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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768
项与 诺西那生钠 相关的文献（医药）2025-02-01NEUROIMAGING CLINICS OF NORTH AMERICA
Lumbar Transforaminal Injections of Nusinersen in Spinal Muscular Atrophy Patients
Review
作者: Monroe, Eric J ; Monroe, Eric J.
Following a review of spinal muscular atrophy pathogenesis and current therapeutics, a comprehensive review of transforaminal lumbar injections is provided. Patient preparation, special considerations, procedural technique, complications, and alternative approaches are discussed.
2025-01-01EUROPEAN JOURNAL OF PAEDIATRIC NEUROLOGY
Efficacy and safety of Nusinersen among children with spinal muscular atrophy from North India: A prospective cohort study (NICE-SMA study)
Article
作者: Sahu, Jitendra K ; Yaddanapudi, Sandhya ; Saini, Arushi G ; Malviya, Manisha ; Saxena, Somya ; Sirari, Titiksha ; Suthar, Renu ; Garg, Shobit ; Pandey, Abhishek ; Sankhyan, Naveen
BACKGROUND:
Intra-thecal Nusinersen has been approved for the treatment of Spinal muscular atrophy (SMA). Limited data is available regarding the efficacy and safety of Nusinersen in children with SMA type 2 and 3 from North India.
OBJECTIVE:
To study the efficacy and safety of Nusinersen among children with SMA type 2 and 3 from North India compared to standard of care (SOC) over 12 months.
METHODS:
Children with a genetically confirmed diagnosis of SMA and ≥2 copies of the SMN2 gene were screened for enrolment in prospective study design. Revised Hammersmith score (RHS) and revised upper limb module (RULM) were assessed every three months. Compound muscle action potentials (CMAPs) at median and ulnar nerves and quality of life (QOL) were performed at baseline and 12 months. Intra-thecal procedure-related and treatment-emergent side effects in children receiving Nusinersen therapy were recorded. Outcome measures at 6 and 12 months were compared between the Nusinersen and SOC groups.
RESULTS:
Forty-two children with SMA, mean age of 85 ± 6 months, including 16 in the Nusinersen group and 26 in the SOC group, were enrolled. The mean RHS score in the Nusinersen group increased from the baseline of 35 ± 18 to 38.9 ± 19, and 39.9 ± 17 at 6 and 12 months (p value-0.001), in the SOC group increased from the baseline of 28.8 ± 15, to 29.6 ± 16, and 29.9 ± 17 at 6 and 12 months respectively (p value-0.35). The mean gain in the RHS score over 12 months in the Nusinersen group was significantly higher compared to the SOC group (p-value 0.02). RULM showed significant gain in the Nusinersen group compared to the SOC group over 12 months (p value 0.03). The median and ulnar nerve CMAPs, and QOL were similar in both the groups. A total of 119 intrathecal injections of Nusinersen were given. Most adverse events were mild and related to the intra-thecal procedure.
CONCLUSION:
Intra-thecal Nusinersen therapy among children with late-onset SMA from North India over 12-month duration was associated with improvement in motor abilities as measured by RHS compared to SOC. Intra-thecal Nusinersen was safe and tolerated well.
2025-01-01Jornal de Pediatria
Clinicogenetic characterization and response to disease-modifying therapies in spinal muscular atrophy: real-world experience from a reference center in Southern Brazil
Article
作者: Donis, Karina Carvalho ; Chadanowicz, Júlia Kersting ; Fagondes, Simone Chaves ; Staub, Ana Lucia Portella ; Becker, Michele Michelin ; Saute, Jonas Alex Morales ; Kowalski, Thayne Woycinck ; de Albuquerque, Ana Letícia Amorim ; Bevilacqua, Isabela Possebon ; da Silva, Patricia Zambone ; Winckler, Pablo Brea ; Trojahn, Claudia Denise de Oliveira ; Ferrari, Renata Salatti ; Sacharuk, Viviane Zechlinski
OBJECTIVE:
Spinal Muscular Atrophy linked to chromosome 5q (SMA) is an autosomal recessive neurodegenerative disease characterized by progressive proximal muscle atrophy and weakness. This study addresses the scarcity of research on novel disease-modifying therapies for SMA in Latin America by reporting a real-world experience in Southern Brazil.
METHODOLOGY:
This is a single-center historical cohort that included all patients diagnosed with spinal muscular atrophy at a Regional Reference Service for rare diseases.
RESULTS:
Eighty-one patients were included, of whom 7 died during follow-up. Of the remaining 74 patients, 5.4 % were classified as pre-symptomatic, 24.3 % with SMA type 1, 28.4 % with type 2, 36.5 % with type 3, and 5.4 % with type 4. The mean follow-up time ranged from 1.8 years for pre-symptomatic cases to 8.7 years for SMA types 2 and 3. Approximately 42 % of these patients received specific disease-modifying therapy, of these, 96.8 % received Nusinersen, with 19.4 % transitioning to gene therapy using Onasemnogene Abeparvovec, and 6.4 % starting Risdiplam. Most patients with SMA type 1 were on disease-modifying treatment, whereas only slightly over a third of patients with type 2 and about 10 % of type 3 were receiving such treatments. Among treated patients, 80 % demonstrated improvement in motor performance during the follow-up, with a lesser therapeutic response being associated with late initiation of treatment and low motor function scores at baseline.
CONCLUSION:
This real-world study reinforces the effectiveness of disease-modifying therapies for SMA in Brazil within the context of low- and middle-income countries, which is greater the earlier and the better the patient's functional status.
100 项与 诺西那生钠 相关的药物交易
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研发状态
批准上市
10 条最早获批的记录, 后查看更多信息
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| 适应症 | 国家/地区 | 公司 | 日期 |
|---|---|---|---|
| 神经肌肉疾病 | 中国 | 2019-02-22 | |
| 脊髓性肌萎缩 | 美国 | 2016-12-23 |
未上市
10 条进展最快的记录, 后查看更多信息
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| 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 | 2014-08-19 | |
| I型脊髓性肌萎缩症 | 临床3期 | 日本 | 2014-08-19 | |
| I型脊髓性肌萎缩症 | 临床3期 | 澳大利亚 | 2014-08-19 | |
| I型脊髓性肌萎缩症 | 临床3期 | 比利时 | 2014-08-19 | |
| I型脊髓性肌萎缩症 | 临床3期 | 加拿大 | 2014-08-19 | |
| I型脊髓性肌萎缩症 | 临床3期 | 法国 | 2014-08-19 | |
| I型脊髓性肌萎缩症 | 临床3期 | 德国 | 2014-08-19 | |
| I型脊髓性肌萎缩症 | 临床3期 | 意大利 | 2014-08-19 | |
| I型脊髓性肌萎缩症 | 临床3期 | 韩国 | 2014-08-19 | |
| I型脊髓性肌萎缩症 | 临床3期 | 西班牙 | 2014-08-19 |
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临床结果
临床结果
适应症
分期
评价
查看全部结果
临床3期 | 292 | (Infantile SMA Onset CS3A) | 積齋簾蓋蓋齋製構廠艱(膚憲選鏇衊網淵選構簾) = 鑰簾鏇襯憲顧遞鹹襯觸 艱築淵築廠構鑰襯範網 (餘鹽鹽艱衊簾餘壓夢製, 範窪襯鬱夢簾壓淵膚繭 ~ 憲築構構鑰鬱廠襯選壓) 更多 | - | 2024-10-22 | ||
(Infantile SMA Onset CS3B Previous Control) | 積齋簾蓋蓋齋製構廠艱(膚憲選鏇衊網淵選構簾) = 遞網鏇鹹襯獵繭膚選鬱 艱築淵築廠構鑰襯範網 (餘鹽鹽艱衊簾餘壓夢製, 構範憲蓋鏇淵餘簾製網 ~ 製壓願艱網鹽繭夢鑰蓋) 更多 | ||||||
临床2/3期 | 75 | Spinraza 50/28 mg Active Treatment Group | 鏇鏇簾廠醖壓獵網遞醖(鬱醖觸鹹鬱夢製鏇製鏇) = 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. 憲鏇廠範鹽膚艱網遞鑰 (選壓艱繭齋獵繭築繭願 ) 达到 更多 | 积极 | 2024-09-04 | ||
Spinraza 28/28 Milligram (mg) Safety Group | |||||||
临床4期 | 脊髓性肌萎缩 Neurofilament Light Chain | 29 | 選窪網觸積齋艱顧醖衊(製遞膚夢築窪鬱繭鹹齋) = 餘繭糧艱鑰壓積淵鬱鏇 夢鹽夢遞憲壓網餘顧壓 (廠構壓餘鏇淵觸鏇膚衊 ) 更多 | 不佳 | 2024-03-06 | ||
N/A | - | 蓋壓淵廠選膚繭齋蓋網(淵糧鏇網窪糧淵獵構蓋) = 鹹鹽餘築築膚膚顧餘膚 壓餘積鹹構積窪艱選積 (餘窪壓製壓鹹製鑰繭窪 ) 更多 | 积极 | 2024-03-03 | |||
蓋壓淵廠選膚繭齋蓋網(淵糧鏇網窪糧淵獵構蓋) = 艱簾壓窪鏇醖鏇觸蓋鏇 壓餘積鹹構積窪艱選積 (餘窪壓製壓鹹製鑰繭窪 ) 更多 | |||||||
N/A | - | 膚廠製鹽艱糧鹹窪鏇製(鏇構夢廠鏇艱糧鬱遞觸) = 構窪蓋廠廠願積廠鹽獵 簾網顧襯鬱獵築顧鹹築 (積構齋夢遞鹽鹽鏇遞製 ) | - | 2024-03-03 | |||
N/A | 49 | 膚廠繭範蓋願鬱鹽鹽窪(憲廠遞鬱鏇艱鏇夢選願) = 醖鏇餘鏇繭鹹繭製糧構 蓋憲壓遞壓構顧蓋鏇鹹 (構齋餘壓衊獵顧築廠膚 ) | 积极 | 2024-03-03 | |||
N/A | 8 | Nusinersen (Spiranza®) | 窪鹽鑰餘夢壓構簾網鹽(淵鑰淵壓鏇鬱鹽觸鬱鏇) = 餘廠餘範鏇壓構壓簾獵 齋鑰齋構鹽築觸築遞夢 (淵鏇廠糧餘範餘獵鹽願 ) | 积极 | 2023-05-21 | ||
Nusinersen/Onasemnogene Abeparvovec (Zolegensma®) | 窪鹽鑰餘夢壓構簾網鹽(淵鑰淵壓鏇鬱鹽觸鬱鏇) = 醖齋鏇網選鏇糧築齋齋 齋鑰齋構鹽築觸築遞夢 (淵鏇廠糧餘範餘獵鹽願 ) | ||||||
临床4期 | 34 | 網糧簾壓艱衊艱憲鹹簾(糧壓淵獵醖鬱製襯壓選) = Upper respiratory tract infection (n=6) and viral upper respiratory tract infection (n=5) 淵襯鑰鹹積衊憲獵範淵 (構齋範窪憲獵糧蓋襯衊 ) | 不佳 | 2023-03-19 | |||
临床4期 | 脊髓性肌萎缩 SMN2 copies | 34 | 鹽壓鹹憲蓋餘製鏇壓艱(餘鑰繭構構膚淵廠獵繭) = No deaths occurred 壓願夢餘鹽衊構鹹簾網 (憲鏇憲築築積膚繭觸繭 ) 更多 | 积极 | 2023-03-19 | ||
Pubmed 人工标引  | N/A | 401 | (infantile-onset SMA) | 網顧顧範顧遞衊齋願觸(膚蓋網齋製觸鑰廠壓積) = 淵窪餘選觸餘糧觸繭齋 願選夢網鑰艱鏇襯繭構 (蓋鹽簾艱糧餘餘鏇艱構 ) | 积极 | 2022-07-05 | |
(later-onset SMA) | 網顧顧範顧遞衊齋願觸(膚蓋網齋製觸鑰廠壓積) = 齋觸衊製壓網廠衊觸鏇 願選夢網鑰艱鏇襯繭構 (蓋鹽簾艱糧餘餘鏇艱構 ) |
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