So, even though the SMN1 transgene is placed inside the cell nucleus, it remains separate there and does not integrate with the human DNA. 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. Decreased platelet counts could occur following infusion with ZOLGENSMA. It has two parts—a gene and a vector—and is made in a laboratory by scientists. ZOLGENSMA is made up of a new, working copy of a human SMN gene that is placed inside a vector. On Friday, May 24, 2019, ZOLGENSMA became the second FDA-approved treatment for Spinal Muscular Atrophy (SMA). Onasemnogen abeparvovek, naprodaj pod trgovskim imenom Zolgensma, je gensko zdravilo, ki se v kombinaciji s kortikosteroidi[2] uporablja za zdravljenje spinalne mišične atrofije (SMA) pri otrocih, mlajših od dveh let. Nevertheless, the majority of treated children tend to improve rapidly due to their natural mobility and the intense physiotherapy that they usually receive. This process happens repeatedly throughout the body with many vectors delivering new SMN genes to motor neuron cells so that SMN protein can be made in those cells. What do I need to know about vaccinations and ZOLGENSMA? US-ZOL-20-0250 07/2020. It also means that people who have previously been in contact with wild-type AAV9 virus might not receive AVXS-101. Patients will receive an oral corticosteroid before and after infusion with ZOLGENSMA and will undergo regular blood tests to monitor liver function. NARRATOR: A dedicated team of scientists has developed a targeted approach for treating spinal muscular atrophy using a one-time-only gene therapy called ZOLGENSMA® (onasemnogene abeparvovec-xioi). As a gene therapy, ZOLGENSMA® (onasemnogene abeparvovec-xioi) is designed to target the genetic root cause of spinal muscular atrophy (SMA) by replacing the function of the missing or nonworking SMN1 gene with a new, working copy of a human SMN gene. This fact makes any redosing of AVXS-101 practically impossible, even if the drug was to lose efficacy over years, as the virus will be instantly neutralised by the body. Around 50% of adults and a small percentage of children naturally have antibodies against AAV9 and cannot receive this gene therapy treatment nor any other treatment that uses AAV9 viruses. What should I watch for before and after infusion with ZOLGENSMA? The genetic modification brought about by this gene therapy does not get passed on to children. AAV viruses also are called non-integrating viruses. Leta 2019 je bilo odobreno v Združenih državah za uporabo v obliki enkratne injekcije v veno, v Evropski uniji pa je zdravilo odobreno od maja 2020. The severity of SMA is related to the number of copies of the SMN2 gene. [8] Formulacijo, ki se injicira v hrbtenični kanal, so proučevali tudi pri bolnikih, mlajših od šestih let, vendar so to raziskavo oktobra 2019 ustavili.[9]. Please see the Full Prescribing Information. This happens throughout the body, with many vectors delivering a new, working copy of the SMN gene to motor neuron cells. As a result, muscles can become so weak that eating, breathing, and moving become difficult, and the disease becomes life threatening. The first clinical trial of AVXS-101 started in May 2015 and included 15 babies with type 1 SMA. If you continue to use this site we will assume that you are happy with it. In October 2019, the company disclosed adverse effects on spinal neurons in lab animals when the treatment was administered intrathecally. It belongs to a class of drugs called gene therapies, or therapies that contain synthetic DNA that partly replaces or adds to the patient’s natural DNA. [2] Pri vztrajnih motnjah delovanja jeter je priporočljiva daljša uporaba kortikosteroidov. ZOLGENSMA drug product, administered to two cohorts of subjects. This website may use cookies to improve the site and your experience. Protection against respiratory syncytial virus (RSV) is recommended. To help you understand how this is possible, let’s look at how ZOLGENSMA works. The vector that delivers the SMN gene is made from a virus called adeno-associated virus 9, or AAV9. 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. These cells control muscle function. EU/UK approval is expected around March 2020. Besedilo se sme prosto uporabljati v skladu z dovoljenjem. 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. Gene therapy targets the genetic root cause of spinal muscular atrophy, or SMA, by replacing the function of the nonworking or missing gene that causes SMA, called the survival motor neuron 1 gene, or SMN1 gene. To make the vector, the DNA of the virus is removed so that the new SMN gene can be put inside. If you have questions about ZOLGENSMA after reading this information, ask your healthcare professional.
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