Decreased platelet counts could occur following infusion with ZOLGENSMA. ZOLGENSMA can cause acute serious liver injury. He graduated from MCPHS University - Boston in 2015 and subsequently completed a PGY-1 Managed Care Pharmacy Residency. zule-ma Zuw-LIY-Maa Z-oo-l-eh-m-ah Record the pronunciation of this word in your own voice and play it to listen to how you have pronounced it. The new gene tells motor neuron cells to produce more survival motor neuron (SMN) protein. Do I need to take precautions with the patient’s bodily waste? In one study, about 91% (20/22) of patients were alive and did not need permanent breathing support at 18 months of age. What should I watch for before and after infusion with ZOLGENSMA? What causes SMA? Protection against respiratory syncytial virus (RSV) is recommended. With each day that passes without treatment, motor neuron cells become weaker and weaker and eventually die. ZOLGENSMA is given as a one-time infusion into a vein. ZOLGENSMA ® (onasemnogene abeparvovec-xioi) is a prescription gene therapy used to treat children less than 2 years old with spinal muscular atrophy (SMA). The Drug List A-Z includes audio pronunciation for many drugs. US-ZOL-20-0250 07/2020, ©2020 AveXis, Inc. All rights reserved. de Biase S, Nilo A, Gigli G, Valente M. Investigational therapies for the treatment of narcolepsy. Zolgensma FDA Approval History. ©2020 AveXis, Inc. All rights reserved. ZOLGENSMA can cause acute serious liver injury. Donovan, treated at ~2 months and pictured at 2½ years, has SMA Type 1. If you have questions about ZOLGENSMA after reading this information, ask your healthcare professional. U.S. National Library of Medicine. The OneGene Program™ is a team of highly trained and dedicated people who will support and guide you and your family before, during, and after treatment. ZOLGENSMA is designed to produce SMN protein, which preserves motor neuron cells and, in turn, sustains the muscle function needed for children to survive. Get answers to these questions and many more with this in-depth guide created just for caregivers. The safety information provided here is not comprehensive. How does ZOLGENSMA work in the body? This website is intended for US residents only. This article is the third in a series on difficult to pronounce drug names. ZOLGENSMA is given as a one-time infusion into a vein. National Institutes of Health. What is the most important information I should know about ZOLGENSMA? 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. What should I watch for before and after infusion with ZOLGENSMA? 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. Expert panelists review the causes, diagnostic work-up, management, and emerging therapies inherent in the evolving paradigm of irritable bowel syndrome. Viral respiratory infections before or after ZOLGENSMA infusion can lead to more serious complications. Nitisinone. Talk to the patient’s doctor about any side effects that bother the patient or that don’t go away. Talk with the patient’s doctor to decide if adjustments to the vaccination schedule are needed to accommodate treatment with a corticosteroid. Get these answers and many more in this in-depth guide created just for caregivers. What are the possible or likely side effects of ZOLGENSMA? Zolgensma made headlines earlier this year for being the world’s most expensive medication at a price of $2.1 million. The information provided in this site is intended only for residents of the United States. Timothy O'Shea, MS, PharmD, is a Clinical Pharmacist working at a regional health insurance plan on the east coast. This website may use cookies to improve the site and your experience. Zolgensma; Definition; Zolgensma (onasemnogene abeparvovec-xioi) is a gene therapy used for the treatment of spinal muscular atrophy (SMA). The safety information provided here is not comprehensive. The American Journal of Pharmacy Benefits. 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 is a prescription gene therapy used to treat children less than 2 years old with spinal muscular atrophy (SMA). Reviewed by J.Stewart BPharm.Last updated on May 29, 2019. ©2020 AveXis, Inc. All rights reserved. Please see the Full Prescribing Information. Know what to expect before, during, and after treatment. ZOLGENSMA was not evaluated in patients with advanced SMA. What do I need to know about vaccinations and ZOLGENSMA? /contributor/timothy-o-shea/2019/10/12-difficult-to-pronounce-drug-names-part-3, Copyright Specialty Pharmacy Times 2006-2019, 2 Clarke Drive Suite 100 Cranbury, NJ 08512 P:609-716-7777 F:609-716-9038, https://livertox.nlm.nih.gov/Nitisinone.htm, https://ghr.nlm.nih.gov/condition/adenosine-deaminase-deficiency. Natural history refers to the progression of a disease in a person over time without treatment. We were so happy that today we are making a difference. This website is intended for US residents only. On treatment day, we were excited, we were hopeful. ZOLGENSMA is: A one-time-only dose; Given intravenously (IV) An infusion that takes 60 minutes; Download A Caregiver’s Guide to ZOLGENSMA How to pronounce drug names Drug List A-Z from Drugs.com . Protection against respiratory syncytial virus (RSV) is recommended. 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. His professional interests include pharmacy legislation and managed care pharmacy. ZOLGENSMA was not evaluated in patients with advanced SMA. 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. It targets the underlying genetic cause of spinal muscular atrophy (SMA). Can you pronounce this word better or … This information does not take the place of talking to your healthcare professional about medical conditions and treatments. He completed his M.S.
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