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第一个被批准的治疗巴顿氏病的方法.

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