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#11. Zolgensma - Knowledge Ecology International

Zolgensma is a gene therapy for spinal muscular atrophy (SMA) that was approved by the US FDA in May 2019, at a price of $2.1 million per patient.

#12. FDA committee takes on complex gene therapy safety ...

About one-third of the 500 patients who have received Zolgensma since its approval have experienced a liver-related adverse event, including one case of serious ...

#13. Onasemnogene Abeparvovec for Spinal Muscular Atrophy

The US Food and Drug Administration (FDA) on May 24, 2019 approved ... by the Swiss drugmaker Novartis, has been marketed under the trade name Zolgensma.

#14. Onasemnogene abeparvovec - 維基百科，自由的百科全書

Onasemnogene abeparvovec，商品名為Zolgensma，中文商品名為諾健生®靜脈懸液 ... 美國FDA批准Zolgensma(onasemnogene abeparvovec-xioi)治療兒童脊髓性肌萎縮症.

#15. FDA allows Novartis gene therapy trials to resume after nearly ...

The FDA's approval of Zolgensma is currently only for the severest patients with "Type 1" SMA, which becomes symptomatic soon after birth and ...

#16. Novartis: FDA approves second Zolgensma site - BioProcess ...

The Novartis facility in North Carolina will begin shipping Zolgensma immediately after receiving US FDA commercial licensure approval.

#17. FDA Closes Zolgensma Data Manipulation Case Without Any ...

Although FDA said the manipulated data did not impact the safety or efficacy of Zolgensma, which could remain on the market, Novartis knew of ...

#18. FDA places hold on Zolgensma trials due to safety concerns

Zolgensma was approved​ by the FDA in May of this year as a therapy for children less than two years old with SMA, with Novartis pricing the ...

#19. FDA Ends Zolgensma Data Manipulation Investigation, Places ...

FDA Ends Zolgensma Data Manipulation Investigation, Places No Penalties on Novartis. Published: Apr 01, 2020 By Alex Keown. Novartis_photobyphm.

#20. Gene Replacement Therapy Clinical Trial for Participants With ...

U.S. FDA Resources. Arms and Interventions ... thus forming a double-stranded transgene ready for transcription. Other Name: Zolgensma ...

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

Zolgensma is the first gene therapy to receive FDA approval for the treatment of SMA. Effectiveness. Zolgensma is given as a one-time dose.

#22. Zolgensma Labeling Updated With Additional Safety Information

The Food and Drug Administration (FDA) has approved several revisions to the prescribing information for Zolgensma (onasemnogene ...

#23. 《基因療法》提交全面非臨床毒理數據! FDA 解除諾華(Novartis ...

諾華(Novartis) 的Zolgensma 已經在2019 年5 月上市，是一種被批准透過靜脈注射治療2 歲以下脊髓性肌肉萎縮症(SMA) 1 型患者的基因療法， ...

#24. Zolgensma FDA Tarafından onaylandı - SMA Hastalığı ile ...

ZOLGENSMA FDA TARAFINDAN ONAYLANDI ... Paylaş : FDA (ABD Gıda ve İlaç Dairesi) 2 yaşından küçük çocukların SMA tedavisi için; Zolgensma'yı onayladı. Detaylı ...

#25. Novartis takes a hit as FDA orders a hold on Zolgensma trials ...

The FDA has ordered Novartis to halt enrollment and dosing in a set of trials for Zolgensma, their gene therapy for lethal cases of spinal muscular atrophy.

#26. Cure SMA's Zolgensma FDA Approval Webinar - YouTube

Cure SMA's Zolgensma FDA Approval Webinar. 917 views917 views. Sep 6, 2019. 9. Dislike. Share. Save. Cure SMA. Cure SMA. 1.7K subscribers.

#27. Novartis to launch new Zolgensma trial after FDA lifts restriction

Novartis will launch a new phase three study to expand the use of Zolgensma - the world's most expensive one time therapy at $2.1 million ...

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

U.S. Food and Drug Administration (FDA)-Approved Indications. Zolgensma is indicated for the treatment of pediatric patients less than 2 years of age with ...

#29. Newly Approved Spinal Muscle Atrophy Treatment Zolgensma ...

... a major milestone in the field: approval by the U.S. Food and Drug Administration (FDA) of a gene therapy known as Zolgensma for SMA.

#30. With FDA Nod, Novartis to Explore Zolgensma in Older SMA ...

... the FDA has given Novartis the green light to test its spinal muscular atrophy (SMA) gene therapy, Zolgensma (AVXS-101), in patients ...

#31. FDA Approves First Gene Therapy Treatment for SMA ...

The preclinical work leading to the FDA approval of Zolgensma was conducted by researchers from Nationwide Children's and The Ohio State ...

#32. FDA Calls Out Drug Company for 'Manipulated' Data

Zolgensma is an adeno-associated virus vector-based gene therapy that addresses the genetic root cause of SMA by replacing the defective or ...

#33. How Zolgensma Works to Treat Spinal Muscular Atrophy

Key takeaways: Zolgensma (onasemnogene abeparvovec-xioi) is a type of gene therapy that's FDA-approved to treat a rare genetic condition ...

#34. Novartis Faces Another Setback for Zolgensma as FDA Halts ...

Following the news that the US Food and Drug Administration (FDA) has halted a trial of Novartis's gene therapy Zolgensma due to safety ...

#35. REGENXBIO Announces First FDA Approval of a Gene ...

FDA approval of Novartis' ZOLGENSMA for the treatment of pediatric patients with SMA marks the first regulatory approval based on ...

#36. SMA Gene Therapy | Boston Children's Hospital

... nation to offer an FDA-approved gene therapy for the treatment of spinal muscular atrophy (SMA). Known by the brand name Zolgensma®, formerly AVXS-101, ...

#37. FDA Accepts AveXis' BLA for SMA Drug Zolgensma Under ...

Priority Review status requires the FDA to review the application and make a decision on whether to approve Zolgensma within six months (the ...

#38. FDA: approval of zolgensma was based on manipulated data

In late May, the FDA approved the gene therapy Zolgensma to treat children with a severe form of spinal muscular atrophy, a leading genetic ...

#39. Onasemnogene Abeparvovec - an overview - ScienceDirect ...

Until 2019, Nusinersen/Spinraza® and Zolgensma® (onasemnogene abeparvovec-xioi) are the FDA approved treatment for SMA [59,60]. The FDA approved Nusinersen ...

#40. Zolgensma® (Onasemnogene Abeparvovec-Xioi)

accordance with the United States Food and Drug Administration (FDA) approved Zolgensma labeling; and. 11. Patient will receive Zolgensma intravenously in ...

#41. FDA fast tracks Novartis' SMA gene therapy - PMLiVE

The one-time treatment, now known as Zolgensma, is designed to address the genetic basis of the deadly neuromuscular disease, which is caused by ...

#42. Novartis broke sacred FDA rule: In God we trust, all others ...

The recent flurry of news about the inclusion of faulty data in the regulatory submission for Zolgensma (onasemnogene abeparvovec-xioi), ...

#43. Novartis stands behind Zolgensma® (onasemnogene ...

of Zolgensma. The FDA supports the continued marketing and use of Zolgensma for patients with spinal muscular atrophy (SMA) less than 2 ...

#44. 美FDA批准史上最貴基因療法造福2歲以下的罕病患者 - Taiwan ...

台灣英文新聞／朱明珠綜合外電報導）美FDA上週五（24日）批准一款治療幼兒罕見疾病─脊髓性肌肉萎縮症的新藥Zolgensma，該款新藥一次性療程費用高達 ...

#45. 全球最貴的藥！諾華基因療法單劑要價超過210萬美元

諾華在Zolgensma獲得FDA核准後，立即宣布了價格。該款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 ...

#46. The most expensive drug in the world just got approval from ...

Zolgensma is the second gene therapy approved by the FDA. The first was a Spark Therapeutics eye disease treatment, Luxturna, which also uses a ...

#47. Novartis hits more problems with Zolgensma; studies halted

The US Food and Drug Administration (FDA) has placed a partial hold on spinal muscular atrophy (SMA) clinical trials for the intrathecal ...

#48. Zolgensma - SMA News Today

It was approved by the U.S. Food and Drug Administration (FDA) in May 2019 to treat newborns and toddlers up to age 2 with all types of spinal ...

#49. Bio Roundup: Zolgensma Fallout, Duchenne Redux, Ebola ...

We learned last week that the FDA was investigating Novartis for manipulating animal data related to its $2 million-a-dose gene therapy ...

#50. 產業觀點／基因療法掀起藥業大變革

... 脊髓性肌肉萎縮症新藥ZOLGENSMA，被美國FDA核准上市，210萬美元的定價 ... 因此2018年初，Spark Therapeutics的基因治療藥物LUXTURNA，被美國FDA ...

#51. FDA Approves Ozanimod, Intrathecal Zolgensma Results ...

The FDA previously approved Zolgensma in May 2019 for the treatment of SMA in pediatric patients 2 years or less of age with mutations in the ...

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

Clinical Trial Results. The FDA approval of Zolgensma was based on the STRIVE and START clinical trials. Efficacy was established based on survival, and ...

#53. 美FDA批准史上最貴基因療法藥劑造福2歲以下的罕病患者

美FDA上週五（24日）批准一款治療幼兒罕見疾病─脊髓性肌肉萎縮症（Spinal MuscularAtrophy，SMA）的新藥Zolgensma，該款新藥一次性療程費用高達 ...

#54. Zolgensma (onasemnogene abeparvovec-xioi) - Rare ...

The FDA gave initial approval for Zolgensma in 2019, making it the first approved gene therapy for SMA and the second disease-modifying ...

#55. The US just approved a $2.1 million treatment for a ...

The US just approved a new treatment for a devastating rare genetic disease. · Called Zolgensma, it's a cutting-edge, one-time treatment called a ...

#56. Zolgensma Birmingham, Alabama (AL)

Zolgensma (onasemnogene abeparvovec-xioi) Treatment for Spinal Muscular Atrophy ... According to the FDA, patients of all ages with all types of SMA less ...

#57. Novartis Pledges to Do Things Differently for FDA, After ...

Despite the data manipulation, both Novartis and federal regulators are in support of keeping the treatment on the market. Zolgensma, approved ...

#58. Novartis used faulty data for world's most expensive drug ...

FDA : Drugmaker knew about 'manipulated data' before approval of the ... The FDA approved Zolgensma for children under the age of two with ...

#59. Novartis could face FDA action over Zolgensma 'data ...

The FDA has said Novartis could face legal action, after it emerged that data filed to support approval of its spinal muscular atrophy gene ...

#60. Update: Novartis' Gene Therapy Zolgensma Gets Nod from EU

Update (24/05/2019): Zolgensma, developed by Novartis, has become the first FDA-approved gene therapy for the treatment of spinal muscular ...

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

On an analyst call today Novartis said Zolgensma sales were being ... the US FDA put another roadblock in the way of intrathecal Zolgensma, ...

#62. 諾華Zolgensma三期試驗積極SMA發病前幼童79%可正常發育

一次性基因療法Zolgensma於2019年5月獲美國FDA批准上市，適用於2歲以下SMA兒童的治療，目前標價為治療一次要價212.5萬美元，也仍是目前市場上最貴的藥物。

#63. Some Data Backing Novartis Gene-Therapy Approval ...

Had the agency known about the manipulation, it would have delayed the approval of Zolgensma, Wilson Bryan, the director of the FDA division ...

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

FDA approves Novartis' $2.1 million gene therapy — making it the world's most expensive drug · The FDA approved Novartis' Zolgensma, a one-time ...

#65. FDA decides not to penalize Novartis for Zolgensma data ...

The FDA decided not to impose any penalties on Novartis for a data manipulation scandal involving the world's most expensive drug, Zolgensma ...

#66. Zolgensma Side Effects Center - RxList

Zolgensma (onasemnogene abeparvovec-xioi) is an adeno-associated virus vector-based gene therapy ... You may report side effects to FDA at 1-800-FDA-1088.

#67. FDA says Novartis withheld data problem before gene therapy ...

Zolgensma, costing $2.1 million, was approved in May for children under 2. It's used to great a rare inherited condition, spinal muscular ...

#68. onasemnogene abeparvovec (Zolgensma) | BCBSND

Description. FDA APPROVED INDICATIONS AND DOSAGE 1. Agent, Indication, Dosage. Zolgensma®. (onasemnogene abeparvovec ...

#69. Novartis' new Zolgensma formulation faces delay as US FDA ...

Zolgensma, the world's most expensive medicine priced at $2.1 million, received FDA approval to treat spinal muscular atrophy, or SMA, ...

#70. FDA Focuses on Novartis Delay in Reporting Drug-Test Data ...

The drug, Zolgensma, is used to treat a sometimes fatal form of spinal muscular atrophy in children and costs about $2.1 million for a one-time ...

#71. zolgensma | GeneOnline News - 基因線上

FDA 相隔兩年終於放行！批准諾華高劑量脊髓注射SMA 藥物試驗. 諾華（Novartis）的脊髓性肌肉萎縮症（SMA）藥物Zolgensma 在2019 年獲准於美國上市，是一種透過…

#72. FDA new drug approvals in Q2 2019 - Nature

24 May. Onasemnogene abeparvovec (Zolgensma; Novartis). SMN1 gene therapy. Spinal muscular atrophy ; 10 June. Polatuzumab vedotin (Polivy; Roche).

#73. FDA says Novartis withheld data problem before drug approval

Zolgensma was approved in May for children under 2, becoming the most expensive treatment ever. It's a one-time treatment for a rare inherited ...

#74. Report: FDA looks into time gap between gene therapy data ...

Zolgensma was approved for spinal muscular atrophy in infants on May 24 and is the highest-priced drug ever, with a list price of $2.1 million.

#75. 罕見疾病 脊髓性肌肉萎縮症 - 藥學雜誌電子報148期

Zolgensma 於2019年獲得FDA許可上市，是目前全球最貴的藥物，適用於2歲以下SMA患者，在START試驗中，接受Zolgensma治療的患者其運動能力，如頭部控制和無需 ...

#76. The problem of manipulated data - Pharmaceutical Technology

In August, the FDA accused Novartis of submitting a drug application for Zolgensma, which included manipulated data, and failing to inform ...

#77. FDA Approves Zolgensma, first gene therapy for SMA - ST ...

Zolgensma, a first gene therapy for spinal muscular atrophy - and first for any chronic neurologic disease - is now an approved and potential "one-time" ...

#78. 諾華SMA基因治療獲美核准，單次療程創天價

上週五美國FDA核准了Zolgensma，被諾華稱為能改變終身的基因療法，該藥用於2歲以下罹患罕疾脊髓性肌肉萎縮症的兒童，全美每年約有400名嬰兒罹患此病， ...

#79. Novartis' Zolgensma (onasemnogene abeparvovec-xioi ...

Novartis' Zolgensma (onasemnogene abeparvovec-xioi) Receives FDA's Approval for Spinal Muscular Atrophy in Pediatric Patients.

#80. Durbin, Baldwin, Sanders, Warren, Blumenthal To FDA

On May 24, 2019, the FDA approved Zolgensma for children with a severe form of spinal muscular atrophy, which is an important medical ...

#81. Zolgensma数据：FDA的“重轻便”警告产业，而不仅仅是诺华

美国FDA可能它处理Zolgensma数据操作的启示的方式取得了诺华的例子联盟再生医学说。 美国食品和药物管理局后一个月（FDA）批准了一次性基因疗法佐根司 ...

#82. Novartis delayed notifying about gene therapy data ... - The BMJ

AveXis learnt of the manipulation this March, the FDA said. Zolgensma was approved on 24 May. Only on 28 June did the company notify the FDA ...

#83. ICER Comments on the FDA Approval of Zolgensma for the ...

ICER Comments on the FDA Approval of Zolgensma for the Treatment of Spinal Muscular Atrophy. –ICER's update reflects the gene therapy's new clinical data ...

#84. FDA Approves Gene Therapy With $2.1M Price Tag for Spinal ...

“Zolgensma is a historic advance for the treatment of SMA and a landmark 1-time gene therapy. Our goal is to ensure broad patient access to this ...

#85. 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 ...

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

While Zolgensma isn't the first gene therapy approved by the FDA, it is held out to be an early test case for the curative promise, and is also ...

#87. FDA Approves Zolgensma, Landmark AAV-Delivered Gene ...

Zolgensma (Novartis, AveXis), an AAV-delivered gene therapy used to treat spinal muscular atrophy (SMA) type 1 also known as AVXS-101, ...

#88. World's priciest drug approved after "data manipulation," FDA ...

The FDA approved Zolgensma on May 24, only to learn a month later of a "data manipulation issue that impacts the accuracy of certain data ...

#89. Gene therapy goes CNS with Zolgensma FDA OK - BioWorld

In late May, Novartis AG's Avexis Inc. unit gained FDA approval for Zolgensma (onasemnogene neparvovec) to treat spinal muscular atrophy, ...

#90. How Parents Get Zolgensma, the World's Most Expensive Drug

Gene therapy Zolgensma is the world's most expensive medicine. ... Novartis AG set the price at $2.1 million after the FDA approval came ...

#91. FDA APPROVAL: Gene Therapy Comes of Age - Pediatrics ...

On May 24, 2019, the FDA approved Zolgensma (formerly AVXS-101), a first-of-its-kind gene therapy for spinal muscular atrophy.

#92. UF Health patient first to receive new gene therapy for ...

The U.S. Food and Drug Administration approved Zolgensma on May 24. ... “I just got a call and the FDA approved the gene therapy today, ...

#93. Novartis Manipulated Zolgensma Data: Fda | Mint

The FDA said the Swiss drug maker was aware of the data problem on 14 March, but did not inform the regulator for more than a month · Zolgensma, ...

#94. New Drug Overview

Zolgensma (onasemnogene abeparvovec-xioi; referred to as ... May 2019, is the second FDA-approved product for the treatment of SMA (FDA ...

#95. Novartis CEO Defends Company's Decision to Withhold False ...

Novartis and the F.D.A. have said the falsified data did not affect the safety, quality or efficacy of Zolgensma, a therapy that treats a ...