p; 6.5 2 3 5 71894-127-05
6.6 - 7.0 1 4 5 71894-128-05
7.1 - 7.5 0 5 5 71894-129-05
7.6 - 8.0 2 4 6 71894-130-06
8.1 - 8.5 1 5 6 71894-131-06
8.6 - 9.0 0 6 6 71894-132-06
9.1 - 9.5 2 5 7 71894-133-07
9.6 - 10.0 1 6 7 71894-134-07
10.1 - 10.5 0 7 7 71894-135-07
10.6 - 11.0 2 6 8 71894-136-08
11.1 - 11.5 1 7 8 71894-137-08
11.6 - 12.0 0 8 8 71894-138-08
12.1 - 12.5 2 7 9 71894-139-09
12.6 - 13.0 1 8 9 71894-140-09
13.1 - 13.5 0 9 9 71894-141-09
小瓶标称浓度为2.0×1013vg/mL，含有不小于5.5mL的可萃取体积。
b样品瓶标称浓度为2.0×1013vg/mL，可萃取体积不小于8.3mL。
存储和处理
•产品在透明小瓶中装运并冷冻（≤-60°C [-76°F]）。
•收到后，立即将试剂盒放入2°C至8°C（36°F至46°F）的冰箱中。
•ZOLGENSMA在2°C至8°C（36°F至46°F）的温度下保存14天。
•请勿重新激活。
•必须在收到后14天内使用。
完整说明资料附件：
https://www.avexis.com/content/pdf/prescribing_information.pdf
FDA approval for Zolgensma®, the first and only gene therapy for pediatric patients with spinal muscular atrophy (SMA)
US Food and Drug Administration (FDA) has approved Zolgensma® (onasemnogene abeparvovec-xioi) for the treatment of pediatric patients less than 2 years of age with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene. Zolgensma is designed to address the genetic root cause of SMA by providing a functional copy of the human SMN gene to halt disease progression through sustained SMN protein expression with a single, one-time intravenous (IV) infusion. Zolgensma is the first and only gene therapy approved by the FDA for the treatment of SMA, including those who are pre-symptomatic at diagnosis.
"A diagnosis of SMA is devastating, leaving untreated babies who have the most severe form with painfully short, highly medicalized lives, during which they are unable to lift their heads, sit or roll, have difficulty swallowing and breathing and need 24-hour care," said Jerry Mendell, M.D., principal investigator at the Center for Gene Therapy at The Abigail Wexner Research Institute of Nationwide Children's Hospital in Columbus, OH. "In the START clinical trial we conducted with Zolgensma, all children were alive at the conclusion of the study and many were able to sit, roll, crawl, play and some could walk. This level of efficacy, delivered as a single, one-time therapy, is truly remarkable and provides a level of unprecedented hope for families battling SMA Type. We now have data four years out from the trial, and we see the durability of this gene therapy."
"The approval of Zolgensma is a testament to the transformational impact gene therapies can have in reimagining the treatment of life-threatening genetic diseases like spinal muscular atrophy," said Vas Narasimhan, CEO of Novartis. "We believe Zolgensma could create a lifetime of possibilities for the children and families impacted by this devastating condition."
SMA is a rare, genetic neuromuscular disease caused by a defective or missing SMN1 gene. Without a functional SMN1 gene, infants with SMA lose the motor neurons responsible for muscle functions such as breathing, swallowing, speaking and walking.
Left untreated, muscles become progressively weaker.
In the most severe form, this eventually leads to paralysis and ultimately perman |
