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#11. ZOLGENSMA® (onasemnogene abeparvovec-xioi) for ...

One-time-only spinal muscular atrophy (SMA) treatment for pediatric patients less than 2 years of age. See Boxed Warning and Full Prescribing Information.

#12. 全球最貴的藥！諾華基因療法單劑要價超過210 萬美元 - 科技新報

諾華在Zolgensma 獲得FDA 核准後，立即宣布價格。此藥劑是治療遺傳型神經肌肉疾病的創新療法，這種疾病具有相當破壞性，肌肉會喪失機能，可能導致兒童在 ...

#13. First patient dosed with Zolgensma in UK lays path for novel ...

Zolgensma is a single-dose adeno-associated viral vector (AAV) gene therapy indicated for SMA, a condition caused by a defect in the survival ...

#14. Zolgensma® meaningfully improves spinal muscular atrophy ...

Zolgensma is the only gene therapy for SMA and the only SMA treatment designed to directly address the genetic root cause of the disease by ...

#15. Minister for Health announces HSE approval to reimburse ...

The new innovative gene therapy treatment Zolgensma was granted EU marketing authorisation for treating SMA in May 2020. The joint process for ...

#16. Zolgensma (onasemnogene abeparvovec) dosing, indications ...

Medscape - Spinal muscular atrophy type 1 disease dosing for Zolgensma (onasemnogene abeparvovec), frequency-based adverse effects, ...

#17. Zolgensma 相關報導- Yahoo奇摩新聞

最新最豐富的Zolgensma 相關新聞就在Yahoo奇摩新聞，讓你快速掌握世界大事、財經動態、體育賽事結果、影劇圈內幕、社會萬象、台灣在地訊息。

#18. NHS to use world's most expensive drug to treat spinal ...

Zolgensma, which costs £1.79m for one-off treatment, will be available in England this year for the first time.

#19. European Study Confirms Safety and Efficacy of Zolgensma in ...

Zolgensma was approved by the FDA in May 2019 for patients less the two years of age with mutations of the SMN1 gene. The therapy provides a ...

#20. Zolgensma / onasemnogene abeparvovec /AVXS-101 - Spinal ...

Zolgensma / onasemnogene abeparvovec /AVXS-101. SMA UK provide a range of information and support to families and health professionals as ...

#21. SMA patients can switch from Spinraza to Zolgensma - Korea ...

In Korea, Zolgensma is indicated for SMA patients with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene or three or fewer copies ...

#22. Zolgensma: Marley gets 'miracle drug' for rare disorder - BBC

Zolgensma : Marley gets 'miracle drug' for rare disorder ... A boy with a rare genetic condition has finally received NHS treatment using a " ...

#23. Zolgensma® (Onasemnogene Abeparvovec-Xioi)

Zolgensma is proven and medically necessary for one treatment per lifetime for the treatment of spinal muscular atrophy. (SMA) in patients who meet all of ...

#24. Zolgensma | SMA Europe

Zolgensma ™ (onasemnogene abeparvovec-xioi) is the second treatment approved for SMA in Europe and the first gene therapy for the condition.

#25. Early Cost-Effectiveness of Onasemnogene Abeparvovec-xioi ...

Early Cost-Effectiveness of Onasemnogene Abeparvovec-xioi (Zolgensma) and Nusinersen (Spinraza) Treatment for Spinal Muscular Atrophy I in The Netherlands With ...

#26. Zolgensma 2 x 10Exp13 vector genomes/mL solution for ...

It is a non-replicating recombinant adeno-associated virus serotype 9 (AAV9) based vector containing the cDNA of the human SMN gene under the control of the ...

#27. How Are Insurers Treating the $2M Drug, Zolgensma? - Policy ...

Zolgensma is meant to be a one-time curative treatment for spinal muscular atrophy (SMA), a rare neuromuscular condition that can lead to death ...

#28. Medicijn Zolgensma® voor zeldzame spierziekte SMA alleen ...

Zolgensma ® is een medicijn dat de oorzaak van de erfelijke spierziekte spinale musculaire atrofie (SMA) aanpakt. Dit geneesmiddel houdt een ...

#29. Zolgensma intravenous: Uses, Side Effects, Interactions ...

How to use Zolgensma 2 X 10Exp13 Vg/Ml Intravenous Suspension, Kit Gene Therapy Agents - SMN Protein Deficiency. Consult your pharmacist or physician. Side ...

#30. Zolgensma (Onasemnogene Abeparvovec-xioi Suspension ...

Zolgensma (Onasemnogene Abeparvovec-xioi Suspension for IV Use) may treat, side effects, dosage, drug interactions, warnings, patient labeling, reviews, ...

#31. Zolgensma: Cost, alternatives, uses, how it works, and more

Zolgensma is a brand-name prescription drug used to treat certain types of spinal muscular atrophy. Learn about costs, alternatives, how it ...

#32. Zolgensma Provides No Proven Benefit Over Spinraza In SMA ...

IQWiG, Germany's health technology appraisal institute, has found that Novartis's gene therapy Zolgensma offers no additional benefit ...

#33. ZOLGENSMA Dosage & Rx Info | Uses, Side Effects - eMPR.com

Miscellaneous musculoskeletal disorders: Indications for: ZOLGENSMA. Treatment of pediatrics <2yrs of age with spinal muscular atrophy (SMA) with bi-allelic ...

#34. Discovering Zolgensma for SMA: What goes into making the ...

It's the most expensive medication on the market!! Zolgensma is a single dose medication to relieve the symptoms of Spinal Muscular Atrophy (SMA) ...

#35. Zolgensma: Baby with spinal disorder recovering well after ...

Baby Marley became one of the first 30 to receive the newly licenced US gene therapy drug, Zolgensma, at Sheffield Children's Hospital on ...

#36. Novartis applauds Quebec, the first province to list Zolgensma

Zolgensma is a gene therapy designed to address the genetic root cause of the disease by replacing the missing or defective SMN1 gene 1. It is ...

#37. Novartis closes U.S. gene therapy site as Zolgensma sales fell ...

Sales of Zolgensma, a one-time treatment for spinal muscular atrophy, were $254 million in the fourth quarter from about $291 million in the ...

#38. 'Anchoring' was at work in setting the price of Zolgensma - Stat ...

Zolgensma is a cure for Type 1 patients, unlike Spinraza (for Type 2). It is easy to point fingers over an absolute number, but it is also ...

#39. Zolgensma Advanced Patient Information - Drugs.com

Uses for Zolgensma. Onasemnogene abeparvovec-xioi injection is used to treat spinal muscular atrophy (SMA) with bi-allelic mutations in the ...

#40. Zolgensma® / Minnesota Department of Human Services

Drug - Zolgensma® (onasemnogene abeparvovec-xioi) [AveXis Inc.] January 2020. Therapeutic area - Spinal Muscular Atrophy (SMA). Approval ...

#41. Long-term Zolgensma data backs up gene therapy's use in ...

Novartis' gene therapy Zolgensma helped keep children diagnosed with spinal muscular atrophy prior to symptoms alive and without need of ...

#42. FDA Places Partial Clinical Hold on Zolgensma Trial Over ...

Zolgensma has a list price of $2.1 million for the one-and-done treatment. It has been approved for all types of spinal muscular atrophy. The ...

#43. FDA committee takes on complex gene therapy safety questions

Two gene therapies were examined in an FDA presentation: Novartis' Zolgensma for spinal muscular atrophy and Spark Therapeutics' Luxturna ...

#44. She's 14 months old and needs a drug that costs $2.1 ... - CNN

... who has a rare genetic disease. The gene therapy she needs, Zolgensma, has been called the world's most expensive drug.

#45. 全球首个脊髓性肌萎缩症(SMA)基因疗法！诺华Zolgensma在 ...

来自已完成的3期STR1VE-EU试验的数据表明，接受Zolgensma治疗后患者运动功能迅速改善，大多数患者达到了1型SMA疾病自然史中未观察到的运动里程碑。安全性 ...

#46. The 'most expensive drug in the world', how it works - The ...

Now there are two recently licensed drugs to treat this devastating affliction. There is a catch, of course. One of these drugs, Zolgensma, ...

#47. Zolgensma® data including patients with more severe SMA at

More than 600 patients now treated with Zolgensma, including some more ... Zolgensma clinical trials for the treatment of patients with SMA.

#48. Zolgensma: The $2.1 Million Drug

It is not a treatment, it is a cure, and a new benchmark in gene therapy. In May, Zolgensma, developed by Illinois-based AveXis, a Novartis ...

#49. Outcome of joint negotiations for Zolgensma | BeNeLuxA

Belgium, Ireland and the Netherlands have reached an agreement on the pricing of Zolgensma, a gene therapy for the treatment of Spinal ...

#50. FDA-Approved Gene Therapies | Zolgensma for SMA - Boston ...

Known by the brand name Zolgensma®, formerly AVXS-101, this gene therapy prevents further motor neuron and muscle degeneration by replacing the defective or ...

#51. Gene therapy's next installment | Nature Biotechnology

Zolgensma is a one-time, intravenous injection of human survival motor neuron gene 1 (SMN1) under the control of chicken beta-actin promoter ...

#52. and payers' blessing – for $2m gene therapy Zolgensma

Novartis has gained FDA approval for its new rare disease gene therapy Zolgensma, unveiling a price of $2.12m per patient, making it the ...

#53. Novartis' gene therapy Zolgensma will cost $2.1 million - CNBC

The therapy, Zolgensma, is a one-time treatment for spinal muscular atrophy — a muscle-wasting disease and leading genetic cause of infant ...

#54. onasemnogene abeparvovec | CADTH

Zolgensma is an adeno-associated virus (AVV) vector-based gene therapy indicated for the treatment of pediatric patients less than 2 years of age with ...

#55. News Feature: Gene therapy successes point to better therapies

The drug Zolgensma was recently in the news for all the wrong reasons. ... of Zolgensma, a new treatment for spinal muscular atrophy (SMA).

#56. Onasemnogene Abeparvovec-xioi (Zolgensma®) - Cigna

This policy supports medical necessity review for onasemnogene abeparvovec-xioi (Zolgensma®). Coverage Policy. Gene Therapy coverage varies across plans.

#57. Onasemnogene abeparvovec for treating spinal muscular ...

Evidence-based recommendations on onasemnogene abeparvovec (Zolgensma) for treating spinal muscular atrophy in babies.

#58. Zolgensma | Therapeutic Goods Administration (TGA)

Zolgensma (onasemnogene abeparvovec) is a non-replicating recombinant adeno-associated viral (AAV) vector that utilises the AAV9 capsid to ...

#59. Novartis wins approval for $2.1m gene therapy Zolgensma

Zolgensma, a single-dose, one-time gene therapy has been approved to treat children less than two years of age with spinal muscular atrophy (SMA) ...

#60. Zolgensma (onasemnogene abeparvovec-xioi) | CenterWatch

Zolgensma (onasemnogene abeparvovec-xioi) is an adeno-associated virus vector-based gene therapy. Zolgensma is specifically indicated for the treatment of ...

#61. How can a drug cost $2.1 million? - SWI swissinfo.ch

Zolgensma treats spinal muscular atrophyExternal link, a deadly disease caused by a genetic defect that weakens a person's muscles so ...

#62. An updated cost-utility model for onasemnogene abeparvovec ...

Background: Recent cost-utility analysis (CUA) models for onasemnogene abeparvovec (Zolgensma®, formerly AVXS-101) in spinal muscular atrophy type 1 (SMA1) ...

#63. This New Treatment Could Save the Lives of Babies. But It ...

The therapy, to be sold as Zolgensma, alters the underlying genetic cause of spinal muscular atrophy and may permanently stop the disease.

#64. Zolgensma demonstrates 'remarkable' long-term results for ...

Ongoing follow-up in the START phase 1 study supported “the long-term favorable safety profile” of Zolgensma in children aged up to 6 years ...

#65. Health Insurance Companies Deny Kids With SMA Gene ...

The therapy, called Zolgensma, is a cutting-edge new type of "gene therapy" treatment that treats the disease at the genetic level.

#66. Zolgensma (onasemnogene abeparvovec) - Maryland ...

Zolgensma (onasemnogene abeparvovec-xioi) is an adeno-associated virus vector-based gene therapy indicated for the treatment of pediatric patients less than ...

#67. Health Canada approves $2.8M treatment for spinal muscular ...

Zolgensma is a one-time dose gene therapy that is approved for SMA and would replace the faulty gene at the root of the disorder. Read more: 2 ...

#68. Onasemnogene abeparvovec (Zolgensma®). HTA ID: 20021

Onasemnogene abeparvovec (Zolgensma®) is indicated for the treatment of patients with 5q spinal muscular atrophy (SMA) with a bi-allelic mutation in the ...

#69. Single-Dose Gene-Replacement Therapy for Spinal Muscular

BackgroundSpinal muscular atrophy type 1 (SMA1) is a progressive, monogenic motor neuron disease with an onset during infancy that results ...

#70. ZOLGENSMA - Orphanet

ZOLGENSMA. Adeno-associated viral vector serotype 9 containing the human SMN gene. Marketing authorization with orphan designation - Europe. Diseases list.

#71. Explained: Why world's most expensive drug Zolgensma costs ...

Zolgensma treats rare genetic disorder Spinal Muscular Atrophy and is touted as the most effective drug for the disorder.

#72. Direct Healthcare Professional Communication - GOV.UK

Zolgensma (onasemnogene abeparvovec) is indicated for the treatment of spinal muscular atrophy (SMA).The overall cumulative exposure is ...

#73. Novartis to Open New Trial of Zolgensma for Older SMA Patients

Novartis, at the FDA's request, will open a pivotal study of Zolgensma, given to older SMA patients via spinal canal injection.

#74. ZOLGENSMA (onasemnogene abeparvovec) - Haute Autorité ...

ZOLGENSMA (onasemnogene abeparvovec) est un traitement de première intention, au même titre que SPINRAZA (nusinersen), à utiliser chez les ...

#75. NHS England strikes deal on life-saving gene-therapy drug ...

In studies Zolgensma, manufactured by Novartis Gene Therapies, has helped babies to reach milestones such as breathe without a ventilator, sit ...

#76. onasemnogene abeparvovec (Zolgensma) - Scottish ...

onasemnogene abeparvovec (Zolgensma) · patients with 5q SMA with a bi-allelic mutation in the SMN1 gene and a clinical diagnosis of SMA type 1, ...

#77. Zolgensma Shows Safety and Durability of Efficacy in Long ...

Data from a recent study suggest that onasemnogene abeparvovec (Zolgensma; Novartis) has a favorable benefit-risk profile in patients with ...

#78. 全球最昂贵的药物诺华SMA基因疗法Zolgensma纳入英国医保

根据最新消息显示，诺华脊髓性肌萎缩症（SMA）基因疗法Zolgensma已获批纳入英国国家医疗服务体系（NHS）中。

#79. Five-Year Extension Results of the Phase 1 START Trial

AveXis, Inc. ZOLGENSMA® (onasemnogene abeparvovec-xioi). 2021. Accessed April 13, 2021. https://www.fda.gov/media/126109/download.

#80. Zolgensma Birmingham, Alabama (AL)

Zolgensma (onasemnogene abeparvovec-xioi) Treatment for Spinal Muscular Atrophy. Zolgensma is a gene therapy approved by the US Federal Drug Administration ...

#81. Onasemnogene Abeparvovec-xioi (Zolgensma) - Aetna

Zolgensma is indicated for the treatment of pediatric patients less than 2 years of age with spinal muscular atrophy (SMA) with bi-allelic mutations in the ...

#82. 號稱世上最貴日本擬將脊髓性肌萎症藥物定價逾4500萬 - 中央社

日本經濟新聞報導，Zolgensma是由瑞士諾華集團（Novartis）子公司AveXis研發，用來醫治罕見基因疾病脊髓性肌肉萎縮症（Spinal Muscular Atrophy，SMA）。

#83. Zolgensma™ - Children's Health

Zolgensma is a gene replacement therapy for pediatric patients who have spinal muscular atrophy (SMA) type 1. Learn more.

#84. Novartis' Zolgensma shows 'continued benefit' in SMA patients

Novartis Gene Therapies has unveiled new interim data from the ongoing Phase III STR1VE-EU clinical trial showing the benefit of Zolgensma ...

#85. Zolgensma flatlines, but Spinraza isn't the beneficiary | Evaluate

Zolgensma was approved in mid-2019, and its launch marked the point at which Spinraza's US sales started to plummet.

#86. 諾華基因療法Zolgensma數據存疑美國FDA展開調查！

Zolgensma 是用於治療2歲以下，在兩個運動神經元存活基因(SMN)的SMN1等位基因上攜帶突變的脊髓性肌肉萎縮症(SMA)患者。今年5月24日被FDA核准上市，因定價 ...

#87. SMA藥物Zolgensma獲批，基因療法開啓全新時代- NAI 500

周五，美國食品藥品監管局（FDA）批准諾華Novartis（NYSE: NVS）旗下的神經肌肉疾病基因療法Zolgensma幷將在美國上市銷售，這也是第2個獲得FDA批准的 ...

#88. 日本醫保納入：千萬元新藥「Zolgensma」治療脊髓性肌肉萎縮症

Novartis/AveXis的Zolgensma®採用Regenxbio公司NAV®基因輸送技術平台中的AAV9，利用病毒表面的外殼能結合細胞表面的半乳糖，使得AAV9能穿過血腦屏障，透過 ...

#89. Zolgensma: A Remarkable New Treatment, An ICER Analysis ...

In May 2019, Novartis obtained U.S. Food and Drug Administration (FDA) approval for the gene therapy onasemnogene abeparvovec-xioi (Zolgensma).

#90. Zolgensma From Novartis Is The Most Expensive Drug Ever ...

Zolgensma, a new drug approved by the FDA Friday, costs more than $2.1 million. It's made by AveXis, a drugmaker owned by pharmaceutical ...

#91. Is the $2.1 Million Price Tag for Novartis' Zolgensma Ridiculous?

What we're talking about with Zolgensma is a gene therapy that's now approved for use in an indication called spinal muscular atrophy, or SMA.

#92. $2.1 million drug to treat rare genetic disease approved by FDA

The "miracle drug", Zolgensma, is the most expensive ever and will be used to treat children with spinal muscular atrophy, or SMA.

#93. How this gene therapy drug earned its $2.1 million price tag

Last May, the FDA approved Zolgensma for use in patients under two years old. In a single dose, Zolgensma replaces the function of the defective ...

#94. 諾華旗下AveXis的科羅拉多工廠開放將用於生產脊髓性肌萎症的 ...

諾華(Norvatis)是在2018年4月以87億美元收購AveXis，並因此取得Zolgensma的權利，這是一種基因療法，Zolgensma也如願的在2019年拿到FDA藥證，並以200 ...

#95. 一針6022萬元！ 俄罕病兒注射地表最貴藥物 - 自由時報

Zolgensma 是基因替代療法，可以替代患病基因從而使身體恢復健康。 ... 瑞士製藥公司「諾華」新藥Zolgensma，可用來治療罹患罕見「脊髓性肌肉萎縮 ...