کد مقاله کد نشریه سال انتشار مقاله انگلیسی نسخه تمام متن
8343750 1541558 2015 6 صفحه PDF دانلود رایگان
عنوان انگلیسی مقاله ISI
Safety of laronidase delivered into the spinal canal for treatment of cervical stenosis in mucopolysaccharidosis I
موضوعات مرتبط
علوم زیستی و بیوفناوری بیوشیمی، ژنتیک و زیست شناسی مولکولی زیست شیمی
پیش نمایش صفحه اول مقاله
Safety of laronidase delivered into the spinal canal for treatment of cervical stenosis in mucopolysaccharidosis I
چکیده انگلیسی
Enzyme replacement therapy with laronidase (recombinant human alpha-l-iduronidase) is successfully used to treat patients with mucopolysaccharidosis type I (MPS I). However, the intravenously-administered enzyme is not expected to treat or prevent neurological deterioration. As MPS I patients suffer from spinal cord compression due in part to thickened spinal meninges, we undertook a phase I clinical trial of lumbar intrathecal laronidase in MPS I subjects age 8 years and older with symptomatic (primarily cervical) spinal cord compression. The study faced significant challenges, including a heterogeneous patient population, difficulty recruiting subjects despite an international collaborative effort, and an inability to include a placebo-controlled design due to ethical concerns. Nine serious adverse events occurred in the subjects. All subjects reported improvement in symptomatology and showed improved neurological examinations, but objective outcome measures did not demonstrate change. Despite limitations, we demonstrated the safety of this approach to treating neurological disease due to MPS I.
ناشر
Database: Elsevier - ScienceDirect (ساینس دایرکت)
Journal: Molecular Genetics and Metabolism - Volume 116, Issues 1–2, September–October 2015, Pages 69-74
نویسندگان
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