©2023 皇冠体育娱乐场网址. 版权所有.

第一个也是唯一的治疗方法 议员六世.

NAGLAZYME® (galsulfase) is an enzyme replacement therapy for the treatment of mucopolysaccharidosis VI (议员六世). NAGLAZYME provides a recombinant version of the enzyme missing in individuals diagnosed with 议员六世.

Severe and life-threatening allergic reactions can occur during NAGLAZYME (galsulfase) infusions and up to 24 hours after infusion. 过敏反应的典型症状包括休克, 呼吸困难, 喘息, 喉咙肿胀, 还有低血压. Please see the 重要安全资讯 below and 完整的处方信息.

议员六世, 也被称为马罗托-拉米综合征, is an inherited lysosomal storage disorder caused by the deficiency of an enzyme normally required for the breakdown of glycosaminoglycans (GAGs). If the enzyme is not present in sufficient quantities, the normal breakdown of GAGs is incomplete or blocked. The cell is unable to excrete the GAG residues, which then accumulate in the lysosomes of the cell. This accumulation disrupts the cell’s normal functioning and gives rise to the physical manifestations of the disease. Only about 1,100 people in the world are thought to live with 议员六世.

Learn more about 皇冠体育娱乐场网址’s 15-year commitment to advance the standard of care for 议员六世 with the short film “15 Candles.”


指示

NAGLAZYME® (galsulfase) is indicated for patients with mucopolysaccharidosis VI (议员六世; Maroteaux-Lamy Syndrome). NAGLAZYME has been shown to improve walking and stair-climbing capacity.

重要安全资讯

Severe and life-threatening allergic reactions can occur during NAGLAZYME (galsulfase) infusions and up to 24 hours after infusion. 过敏反应的典型症状包括休克, 呼吸困难, 喘息, 喉咙肿胀, 还有低血压. 如果在输液过程中发生严重的过敏反应, the infusion should be stopped immediately and you should receive medical attention. 联系 your doctor or get medical help right away if you develop any severe symptoms after infusion.

In clinical trials, most patients developed antibodies to NAGLAZYME treatment. There was no clear relationship between antibody formation and the safety or effectiveness of NAGLAZYME.

Serious and severe infusion reactions are associated with NAGLAZYME, 包括荨麻疹, 胸部疼痛, 皮疹, 腹部疼痛, 呼吸困难, 肿胀, 发热, 眼睛刺激. You should receive medication such as antihistamines before NAGLAZYME infusions to reduce the risk of infusion reactions. 如果输注反应发生, the infusion should be slowed or stopped and you may be given additional medication.

The most common side effects of NAGLAZYME seen in clinical trials were 皮疹, 疼痛, 荨麻疹, 发热, 瘙痒, 发冷, 头疼, 恶心想吐, 呕吐, 腹痛,呼吸困难. The most common side effects requiring medical attention are infusion-related effects.

These are not all of the possible side effects with NAGLAZYME. Talk to your doctor if you have any symptoms that bother you or that do not go away.

NAGLAZYME是处方药. Before treatment with NAGLAZYME, it is important to discuss your medical history with your doctor. Tell your doctor if you are taking any medication and if you are allergic to any medicines. Your doctor will decide if NAGLAZYME is right for you. If you have questions or would like more information about NAGLAZYME, contact your doctor.

Spinal cord damage may occur due to the natural 议员六世 disease process. 脊髓损伤的症状包括背部疼痛, 膀胱和肠道失控, 麻木, 和瘫痪. 联系 your doctor immediately if you develop any of these symptoms.

Call your doctor for medical advice about side effects. You may report side effects to 皇冠体育娱乐场网址 at 1-866-906-6100 and the 皇冠体育官网 by visiting www.食品及药物管理局.gov /提交确证 或致电1-800-皇冠体育官网-1088.

For more information, call 皇冠体育娱乐场网址 Rare连接ions™ at 1-866-906-6100.

请参阅全文 处方信息.


图片:处理烧杯的科学家
皇冠体育娱乐场网址的管道

We maximize the impact of genetic innovation to develop transformative therapies.

Each drug candidate we pursue is guided by a fundamental understanding of the genetics and underlying biology of the condition it will address.

了解更多 关于皇冠体育娱乐场网址的管道