LYRIC: A research Study for Sanfilippo Syndrome Type A Study - TopicsExpress

LYRIC: A research Study for Sanfilippo Syndrome Type A Study Objective: The purpose of this study is to assess the potential clinical efficacy of HGT-1410 administered via a surgically implanted IDDD in patients with MPS IIIA. Clinical Trial Overview: 1. This study will be conducted globally, with some of the following countries being considered: Brazil, Canada, France, Spain, United Kingdom, and United States. 2. This study is designed to evaluate the efficacy and safety of HGT-1410 administered via an IDDD versus no treatment in patients at a relatively early stage of MPS IIIA disease. 3. 18 patients will be randomized to one of three groups: 45 mg HGT-1410 administered IT every 2 weeks (6 patients); 45 mg of HGT-1410 administered IT every 4 weeks (6 patients); or a no treatment control arm (6 patients).

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Thanks Sara Carter for my nomination! Heres my 7 interesting

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UPDATE - We wanted to let everyone know that we have closed

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