Posts tagged with it-idursulfase

Who is Smart Enough for a #RareDisease Clinical Trial?

Posted Tuesday October 14, 2014 by Melissa

The Phase II/III Shire intrathecal idursulfase clinical trial requires a child to...

The Phase II/III Shire intrathecal idursulfase clinical trial requires a child to test between a score of 55 and 85 on the DAS II neurocognitive assessment (essentially, an IQ test)....

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Participating in a Clinical Trial: an Interview with Melissa Thomas

Posted Friday August 29, 2014 by Melissa

Recently I had a conversation with Melissa Thomas, a mom to two...

Recently I had a conversation with Melissa Thomas, a mom to two boys with Hunter Syndrome. Her son Nathan passed away from the effects of the disease about five years...

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Immune Tolerance to Enzyme Replacement Therapies, Part 2: Idursulfase

Posted Tuesday June 17, 2014 by Melissa

In Part 1 of this series, I explored some of the background...

In Part 1 of this series, I explored some of the background concepts related to immunogenicity. Now let’s begin examining immunogenicity in the context of a specific example – idursulfase...

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What is Compelling Evidence? The mother frogs speak

Posted Friday August 23, 2013 by Melissa

What have you been doing for the past 3 1/2 years? Having...

What have you been doing for the past 3 1/2 years? Having a few children? Learning a new language? Starting a new job? I’ve been watching lives being saved. Children...

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