zolgensma | A Gene Therapy That Targets the Root Cause of SMA

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Search Results related to zolgensma on Search Engine

A Gene Therapy That Targets the Root Cause of SMA | ZOLGENSMA
zolgensma.com

https://www.zolgensma.com/

WEBZOLGENSMA ® (onasemnogene abeparvovec-xioi) is an essential one-time treatment option that targets the genetic root cause of spinal muscular atrophy (SMA) by replacing the function of the missing or nonworking SMN1 gene—stopping progression of SMA.

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How ZOLGENSMA® (onasemnogene abeparvovec-xioi) works
zolgensma.com

https://www.zolgensma.com/how-zolgensma-works

WEBZOLGENSMA is a gene therapy for SMA that replaces the function of the missing or nonworking SMN1 gene with a new, working SMN gene. The new SMN gene helps the body’s cells—particularly the motor neuron cells—to keep producing SMN protein. What causes SMA? Missing or nonworking SMN1 gene.

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Onasemnogene abeparvovec - Wikipedia
wikipedia.org

https://en.wikipedia.org/wiki/Onasemnogene_abeparvovec

WEBOnasemnogene abeparvovec, sold under the brand name Zolgensma, is a gene therapy used to treat spinal muscular atrophy (SMA), [6] [7] a disease causing muscle function loss in children. It involves a one-time infusion of the medication into a vein. [6] It works by providing a new copy of the SMN gene that produces the SMN protein.

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Zolgensma: Uses, Dosage, Side Effects, Warnings - Drugs.com
drugs.com

https://www.drugs.com/zolgensma.html

WEBJun 12, 2023 · Zolgensma is a gene therapy used for spinal muscular atrophy (SMA). SMA is an inherited disorder that causes skeletal muscle weakness and wasting, which worsens with age. SMA is caused when a gene, usually SMA1, is missing or doesn't work properly.

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Zolgensma: Cost, alternatives, uses, how it works, and more
medicalnewstoday.com

https://www.medicalnewstoday.com/articles/drugs-zolgensma

WEBMar 24, 2021 · How it works. Dosage. How it’s administered. Side effects. Zolgensma and alcohol. Interactions. Pregnancy. Breastfeeding. FAQs. Precautions. What is Zolgensma? Zolgensma is a brand-name...

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FDA approves innovative gene therapy to treat pediatric patients …
fda.gov

https://www.fda.gov/news-events/press-announcements/fda-approves-innovative-gene-therapy-treat-pediatric-patients-spinal-muscular-atrophy-rare-disease

WEBThe U.S. Food and Drug Administration today approved Zolgensma (onasemnogene abeparvovec-xioi), the first gene therapy approved to treat children less than two years of age with spinal muscular...

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What Is SMA? | ZOLGENSMA® (onasemnogene abeparvovec-xioi)
zolgensma.com

https://www.zolgensma.com/about-sma

WEBZOLGENSMA is a prescription gene therapy used to treat children less than 2 years old with spinal muscular atrophy (SMA). ZOLGENSMA is given as a one-time infusion into a vein. ZOLGENSMA was not evaluated in patients with advanced SMA. The safety information provided here is not comprehensive.

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ZOLGENSMA | FDA - U.S. Food and Drug Administration
fda.gov

https://www.fda.gov/vaccines-blood-biologics/zolgensma

WEBOct 18, 2023 · ZOLGENSMA (onasemnogene abeparvovec-xioi) is an adeno-associated virus vector-based gene therapy indicated for the treatment of pediatric patients less than 2 years of age with spinal muscular...

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Zolgensma | European Medicines Agency
europa.eu

https://www.ema.europa.eu/en/medicines/human/EPAR/zolgensma

WEBMar 27, 2020 · Overview. Zolgensma is a gene therapy medicine for treating spinal muscular atrophy, a serious condition of the nerves that causes muscle wasting and weakness. It is intended for patients with inherited mutations affecting a gene known as SMN1, who have either been diagnosed with SMA type 1 (the most severe type) or have …

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Mechanism of Action | ZOLGENSMA® (onasemnogene …
zolgensma-hcp.com

https://www.zolgensma-hcp.com/about-zolgensma/moa/

WEBMechanism of action. ZOLGENSMA is a gene therapy designed to treat the genetic root cause of SMA 1. Spinal muscular atrophy (SMA) is caused by the deletion or mutation of the survival motor neuron 1 ( SMN1) gene. The SMN1 gene produces survival motor neuron (SMN) protein that is critical for normal function of motor neurons.

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