Life-threatening allergic reactions, known as anaphylaxis, can occur during VIMIZIM® (elosulfase阿尔法)输注. Typical signs of anaphylaxis include cough, 皮疹, 喉咙闷, 荨麻疹, 冲洗, 皮肤颜色的变化, 低血压, 呼吸急促（气促）, 胸部疼痛, and gastrointestinal symptoms such as nausea, 腹部疼痛, 干呕, 和呕吐. 联系 your doctor or get medical help right away if these symptoms occur during or after VIMIZIM infusions. If you have a respiratory illness, you may be at risk for a sudden worsening of your condition, and you may require additional monitoring.
当前的研究
研究110 - 504
一个多中心, 跨国公司, 观察 Morquio A Registry Study in 病人s With Mucopolysaccharidosis IVA (Morquio A Syndrome)
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指示
议员IVA (Mucopolysaccharidosis IVA, also known as Morquio A syndrome)
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临床实验的治疗
没有一个
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研究类型
观察, Non-Interventional Registry
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目标
This 第三阶段 extension study will evaluate the long-term efficacy and safety of BMN 110 2.0 mg/kg/week and/or BMN 110 2.0 mg/kg/every other week in patients with mucopolysaccharidosis IVA (Morquio A Syndrome)
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状态
活跃，不招人
完成研究
研究铁道部- 005
Long-Term Efficacy and Safety Extension Study of BMN 110 in 病人s With Mucopolysaccharidosis IVA (Morquio A Syndrome)
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指示
议员IVA (Mucopolysaccharidosis IVA, also known as Morquio A syndrome)
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临床实验的治疗
Vimizim® (elosulfase alfa或bmn110)
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研究类型
第三阶段
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目标
Evaluate the long-term efficacy and safety of BMN 110 2.0 mg/kg/week and/or BMN 110 2.0 mg/kg/every other week in patients with mucopolysaccharidosis IVA (Morquio A Syndrome)
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状态
This study has been completed
研究bmn - 110
Open-label 第二阶段 study of BMN 110 in Pediatric 病人s < 5 Years of Age With Mucopolysaccharidosis IVA (Morquio A Syndrome)
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指示
议员IVA (Mucopolysaccharidosis IVA, also known as Morquio A syndrome)
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临床实验的治疗
Vimizim® (elosulfase alfa或bmn110)
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研究类型
第二阶段
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目标
Evaluate the safety and efficacy of weekly 2.0 mg/kg/wk infusions of BMN 110 in pediatric patients, less than 5 years of age at the time of administration of the first dose of study drug, diagnosed with 议员IVA (Morquio A Syndrome) for up to 208 weeks
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状态
This study has been completed
研究铁道部- 007
Efficacy and Safety Study of BMN 110 for Morquio A Syndrome 病人s Who Have Limited Ambulation
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指示
议员IVA (Mucopolysaccharidosis IVA, also known as Morquio A syndrome)
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临床实验的治疗
Vimizim® (elosulfase alfa或bmn110)
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研究类型
第二阶段
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目标
评估2的效果.0 mg/kg/week BMN 110 in a patient population that has limited ambulation, in a period of up to 144 weeks
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状态
This study has been terminated
研究铁道部- 001
A Clinical Assessment Study of Subjects With Mucopolysaccharidosis IVA (Morquio Syndrome)
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指示
议员IVA (Mucopolysaccharidosis IVA, also known as Morquio A syndrome)
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临床实验的治疗
Vimizim® (elosulfase alfa或bmn110)
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研究类型
观察
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目标
这个多中心, 跨国公司, longitudinal study will quantify endurance and respiratory function in subjects diagnosed with 议员IVA and will better characterize the spectrum of symptoms and biochemical abnormalities in 议员IVA disease over time
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状态
This study has been terminated
研究铁道部- 100
A Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in 病人s With Mucopolysaccharidosis IVA (Morquio A Syndrome)
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指示
议员IVA (Mucopolysaccharidosis IVA, also known as Morquio A syndrome)
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临床实验的治疗
Vimizim® (elosulfase alfa或bmn110)
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研究类型
第二阶段
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目标
这个多中心, open-label extension study is designed to assess long-term efficacy and safety of 2.0 milligrams (mg)/kilogram(kg)/week of BMN 110 in patients diagnosed with Mucopolysaccharidosis IVA (议员IVA)
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状态
This study has been terminated
研究铁道部- 008
Safety and Exercise Study of BMN 110 for Morquio A Syndrome
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指示
议员IVA (Mucopolysaccharidosis IVA, also known as Morquio A syndrome)
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临床实验的治疗
Vimizim® (elosulfase alfa或bmn110)
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研究类型
第二阶段
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目标
评估一个2的安全性.0 mg/kg/周和4.0 mg/kg/week of BMN 110 in patients with Morquio A syndrome for up to 192 weeks. Secondary objectives will investigate the effect of the two doses on exercise capacity for up to 192 weeks. In addition, the pharmacokinetic (PK) parameters of both doses of BMN 110 will be assessed.
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状态
This study has been terminated