BRINEURA® (cerliponase阿尔法) is indicated to slow the loss of ambulation in symptomatic pediatric patients 3 years of age and older with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2), 也被称为三肽基肽酶1 (TPP1)缺乏. Brineura is the first enzyme replacement therapy to be directly administered into the fluid of the brain, treating the underlying cause of CLN2 disease by helping to replace the deficient TPP1 enzyme missing in affected children.
因为可能有过敏反应, 应随时提供适当的医疗支助, 对患者应密切观察, Brineura输注期间和之后. If anaphylaxis occurs, immediately discontinue infusion and initiate appropriate medical treatment. Inform patients/caregivers of the signs and symptoms of anaphylaxis and to seek immediate medical care should these occur. Consider the risks and benefits of readministration of Brineura following an anaphylactic reaction.
CLN2 disease is an ultra-rare and rapidly progressing brain disorder that affects an estimated 20 children born in the United States each year–less than one in a million Americans.
Learn more about the development of the first therapy for CLN2 disease with “The Race for BRINEURA.”
BRINEURA® (cerliponase阿尔法) is a prescription medication used to slow loss of ability to walk or crawl (ambulation) in symptomatic pediatric patients 3 years of age and older with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2), 也被称为三肽基肽酶1 (TPP1)缺乏.
BRINEURA是一种处方药. BRINEURA治疗前, it is important to discuss your child’s medical history with their doctor. Tell the doctor if they are sick or taking any medication and if they are allergic to any medicines. 您孩子的医生将决定BRINEURA是否适合他们. If you have questions or would like more information about BRINEURA, contact your child’s doctor.
BRINEURA is only given by infusion into the fluid of the brain (known as an intraventricular injection) and using sterile technique to reduce the risk of infection. An intraventricular access device or port must be in place at least 5 to 7 days prior to the first infusion. 脑室内通路装置相关感染, 包括脑膜炎, BRINEURA治疗组的观察结果. If any signs of infection or meningitis occur, contact your child’s doctor immediately. The signs and symptoms of infections may not be readily apparent in patients with CLN2 disease. Your doctor should vigilantly be looking for signs and symptoms of infection, 包括脑膜炎, 在BRINEURA治疗期间.
Your child’s intraventricular access device should be replaced prior to 4 years of single-puncture administration of BRINEURA, 因为设备可能会因反复使用而变质.
BRINEURA should not be used in patients with active intraventricular access device-related complications (e.g., leakage, 设备故障, 或者设备相关感染, 包括脑膜炎), 急性症状, 装置插入部位周围未解决的局部感染(例如.g. cellulitis or abscess), or and with shunts used to drain extra fluid around the brain. Your child’s doctor should inspect the scalp and collect samples of your child’s cerebrospinal fluid (CSF) prior to each infusion of BRINEURA, 检查是否存在设备故障或感染.
Low blood pressure and/or 心率慢 may occur during and following the BRINEURA infusion. 如果发生这些反应，请立即联系您孩子的医生.
与BRINEURA治疗相关的不良或超敏反应, 包括发烧, vomiting, 和易怒, 可能在治疗期间和输液后24小时发生. Your child may receive medication such as antihistamines before BRINEURA infusions to reduce the risk of reactions. 可能发生严重和严重的过敏反应(过敏反应). If a reaction occurs, the infusion will be stopped and your child may be given additional medication. 如果发生严重的反应, the infusion will be stopped and your child will receive appropriate medical treatment. 如果出现任何过敏反应的迹象，立即就医.
Safety and effectiveness in pediatric patients below 3 years of age have not been established.
The most common side effects reported during BRINEURA infusions included fever, 心脏电活动的问题, 脑积水脑液中蛋白质的减少或增加, vomiting, seizures, 超敏反应, 血管外的血液收集(血肿), headache, 易怒, 并且增加了脑液中的白细胞计数, device-related感染, 心率慢, 感到紧张不安, 还有低血压. 脑室内装置相关的副作用包括感染, 分娩系统相关并发症, 脑液中白细胞数量增加.
这些还不是BRINEURA可能产生的全部副作用. Talk to your child’s doctor if they have any symptoms that bother them or that do not go away.
打电话给你的医生咨询有关副作用的医疗建议. 你可以向澳门正规博彩十大排行平台制药公司报告副作用. 拨打1-866-906-6100，或拨打1-800-澳门十大博彩正规游戏平台-1088，或访问 www.fda.gov/medwatch.
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