ZOLGENSMA® (onasemnogene abeparvovec-xioi) is a prescription gene therapy used to treat children less than 2 years old with spinal muscular atrophy (SMA). Seek immediate medical attention if a patient experiences unexpected bleeding or bruising. Targets the genetic root cause of SMA. The new gene tells motor neuron cells to produce more survival motor neuron (SMN) protein. ZOLGENSMA is a prescription gene therapy used to treat children less than 2 years old with spinal muscular atrophy (SMA).

The information provided in this site is intended only for residents of the United States. Zolgensma; Definition; Zolgensma (onasemnogene abeparvovec-xioi) is a gene therapy used for the treatment of spinal muscular atrophy (SMA). US-ZOL-20-0250 07/2020, ©2020 AveXis, Inc. All rights reserved.

What causes SMA? The Drug List A-Z includes audio pronunciation for many drugs. Liver enzymes could become elevated and may reflect acute serious liver injury in children who receive ZOLGENSMA. This website is intended for US residents only. Decreased platelet counts could occur following infusion with ZOLGENSMA. Talk to the patient’s doctor about any side effects that bother the patient or that don’t go away. ZOLGENSMA ® (onasemnogene abeparvovec-xioi) is a prescription gene therapy used to treat children less than 2 years old with spinal muscular atrophy (SMA). Additionally he works per diem at a nationwide retail pharmacy chain. FDA Approved: Yes (First approved May 24, 2019) Brand name: Zolgensma Generic name: onasemnogene abeparvovec-xioi Dosage form: Suspension for Intravenous Infusion Company: Novartis Pharmaceuticals Corporation Treatment for: Spinal Muscular Atrophy Zolgensma (onasemnogene …

Zolgensma (onasemnogene abeparvovec-xioi), previously known as AVXS-101, is a gene therapy initially developed by AveXis, now part of Novartis, which is further developing and marketing the treatment. Talk with the patient’s doctor to decide if adjustments to the vaccination schedule are needed to accommodate treatment with a corticosteroid. Know what to expect before, during, and after treatment. If you have questions about ZOLGENSMA after reading this information, ask your healthcare professional. Contact the patient’s doctor immediately if you see signs of a possible viral respiratory infection such as coughing, wheezing, sneezing, runny nose, sore throat, or fever. Reviewed by J.Stewart BPharm.Last updated on May 29, 2019.

It targets the underlying genetic cause of spinal muscular atrophy (SMA). Get answers to these questions and many more with this in-depth guide created just for caregivers. What are the possible or likely side effects of ZOLGENSMA? What do I need to know about vaccinations and ZOLGENSMA? ©2020 AveXis, Inc. All rights reserved. Liver enzymes could become elevated and may reflect acute serious liver injury in children who receive ZOLGENSMA. You are encouraged to report suspected side effects by contacting the FDA at 1-800-FDA-1088 or www.fda.gov/medwatch, or AveXis at 833-828-3947. US-ZOL-20-0250 07/2020, ©2020 AveXis, Inc. All rights reserved. Seek immediate medical attention if a patient experiences unexpected bleeding or bruising.
ZOLGENSMA is: A one-time-only dose; Given intravenously (IV) An infusion that takes 60 minutes; Download A Caregiver’s Guide to ZOLGENSMA Do I need to take precautions with the patient’s bodily waste? ZOLGENSMA can cause acute serious liver injury. National Institutes of Health. Contact the patient’s doctor immediately if the patient’s skin and/or whites of the eyes appear yellowish, or if the patient misses a dose of the corticosteroid or vomits it up. Zolgensma was approved by the U.S. Food and Drug Administration (FDA) in 2019 to treat all types of SMA in … He can be followed on Twitter at @toshea125. Please see the Full Prescribing Information. Permanent breathing support is defined as tracheostomy or the need for continuous use of a machine to help breathe for at least 16 hours every day for 2 weeks or more in a child who did not have a severe and short-lasting, reversible illness or surgery. Hear about the day they received ZOLGENSMA and see how they are doing years after treatment. Otherwise a phonetic pronunciation is provided. Protection against respiratory syncytial virus (RSV) is recommended. Timothy O'Shea, MS, PharmD, is a Clinical Pharmacist working at a regional health insurance plan on the east coast. Once these cells stop working, they cannot be brought back.

ZOLGENSMA was not evaluated in patients with advanced SMA. What is ZOLGENSMA? What should I watch for before and after infusion with ZOLGENSMA? His professional interests include pharmacy legislation and managed care pharmacy. What causes SMA? It is manufactured by AveXis. Contact the patient’s doctor immediately if the patient’s skin and/or whites of the eyes appear yellowish, or if the patient misses a dose of the corticosteroid or vomits it up. What are the results of the clinical studies? You are encouraged to report suspected side effects by contacting the FDA at 1-800-FDA-1088 or www.fda.gov/medwatch, or AveXis at 833-828-3947. ZOLGENSMA is designed to produce SMN protein, which preserves motor neuron cells and, in turn, sustains the muscle function needed for children to survive. Catch up with some of the children with SMA Type 1 who took part in group 2 of the START clinical study of ZOLGENSMA. This website may use cookies to improve the site and your experience. The safety information provided here is not comprehensive. What are the possible or likely side effects of ZOLGENSMA? While ZOLGENSMA keeps motor neurons working to preserve strength in the muscles, it is not a cure and cannot reverse damage already caused by SMA before treatment. It targets the genetic root cause of SMA with a one-time-only dose and replaces the function of the missing or nonworking survival motor neuron 1 (SMN1) gene with a new, working copy of a human SMN gene. ZOLGENSMA is given as a one-time infusion into a vein. In one study, about 91% (20/22) of patients were alive and did not need permanent breathing support at 18 months of age. How does ZOLGENSMA work in the body? U.S. National Library of Medicine. See how AveXis collects, uses, and shares your personal information in our Privacy Policy. What do I need to know about vaccinations and ZOLGENSMA? This information does not take the place of talking to your healthcare professional about medical conditions and treatments. See how AveXis collects, uses, and shares your personal information in our Privacy Policy. Zolgensma made headlines earlier this year for being the world’s most expensive medication at a price of $2.1 million. What should I watch for before and after infusion with ZOLGENSMA? What is the most important information I should know about ZOLGENSMA?

How to pronounce drug names Drug List A-Z from Drugs.com . US-ZOL-20-0250 07/2020. Learn about the clinical study results in children with SMA. ZOLGENSMA is given as a one-time infusion into a vein. This article is the third in a series on difficult to pronounce drug names. Talk with the patient’s doctor to decide if adjustments to the vaccination schedule are needed to accommodate treatment with a corticosteroid. ZOLGENSMA can cause acute serious liver injury.

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