Posts filed in IT Clinical Trial

Inside a Rare Disease Clinical Trial – #HunterSyndromeLive

Posted Sunday February 22, 2015 by Melissa

Since 2011, once a year, Case Hogan travels to Chapel Hill, NC...

Since 2011, once a year, Case Hogan travels to Chapel Hill, NC for an annual clinical trial visit. It is 11 days of travel, physical testing, cognitive testing, infusions, procedures...

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Inside a Rare Disease Clinical Trial – #HunterSyndromeLive

Forgetting, and not

Posted Wednesday November 12, 2014 by Melissa

Four years ago, we had a book of photos of family members...

Four years ago, we had a book of photos of family members that was used as part of Case’s IEP. We didn’t want him to forget who the people were...

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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 3: FDA/NORD Workshop on the Role of Immune Tolerance Induction

Posted Wednesday August 27, 2014 by Melissa

Immune response is one of the most important issue in enzyme replacement...

Immune response is one of the most important issue in enzyme replacement therapy today. At least it is to many patients. That’s why I’ve waited several months to publish this...

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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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Immune Tolerance to Enzyme Replacement Therapies, Part 1: Background on Immunogenicity

Posted Friday June 13, 2014 by Melissa

It would not surprise me if many of you reading this have...

It would not surprise me if many of you reading this have never heard of immune tolerance induction. That may be, in large part, because prior to the last two...

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Flexibility + Medical Trauma + Miracles = Clinical Trial

Posted Wednesday April 02, 2014 by Melissa

Not all clinical trials are the same – some involve just a...

Not all clinical trials are the same – some involve just a single blood draw, or interview, or procedure. But others, like the MPS II intrathecal trial, are very demanding,...

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#SaveJack, #BeBrave

Posted Friday January 17, 2014 by Melissa

I’ll never feel okay about kids dying. Never. And I’ll surely never...

I’ll never feel okay about kids dying. Never. And I’ll surely never feel okay about my best friend’s son dying. But in the whole convoluted process that is drug discovery,...

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What proof does the FDA want in a degenerative disease?

Posted Thursday January 16, 2014 by Melissa

Here is an example in Duchenne MD of why I spent last...

Here is an example in Duchenne MD of why I spent last week at the FDA. In a degenerative disease, if a drug can maintain abilities, why do children have...

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For Jack

Posted Sunday December 01, 2013 by Melissa

There aren’t really words to describe what Jack means to me. Besides...

There aren’t really words to describe what Jack means to me. Besides Case, Jack was the first boy with Hunter Syndrome that I met. His mom, my friend Jamie, welcomed...

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AIM-IT: Phase III trial for Idursulfase IT

Posted Friday October 25, 2013 by Melissa

I’m sitting at the National MPS Society conference and learning more about...

I’m sitting at the National MPS Society conference and learning more about the next step in the testing of Idursulfase IT – the Phase III trial. I know families are...

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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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A Tale of Two Frogs: a rare disease story

Posted Tuesday July 30, 2013 by Melissa

“It’s a miracle!” she said, as she watched her son hop, ever...

“It’s a miracle!” she said, as she watched her son hop, ever so gently, for the first time. Having never had legs before, even hopping was quite a new endeavor...

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The Phase III

Posted Monday July 01, 2013 by Melissa

Our community has been waiting with anticipation for the next phase of...

Our community has been waiting with anticipation for the next phase of this intrathecal trial to begin, that is, the Phase III clinical trial. Last week, I became aware of...

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Intrathecal Trial Update – “Jeep for me!”

Posted Thursday June 13, 2013 by Melissa

Case hit another milestone by claiming Jeeps...

Case hit another milestone by claiming Jeeps...

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Hunter Syndrome 2.0

Posted Friday December 07, 2012 by Melissa

I had a great conversation a few months ago with my friend...

I had a great conversation a few months ago with my friend Chris Dutcher, an adult with Hunter Syndrome. Now if you’re a friend who may only know Case’s “version”...

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It's a New Day

Intrathecal Trial Update – New port (hopefully)

Posted Wednesday June 27, 2012 by Melissa

So we recently learned about the new port-a-cath that is contemplated to...

So we recently learned about the new port-a-cath that is contemplated to be used in the next phases of the intrathecal clinical trial. If you’ve followed along, you’ll know that...

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Soph-a-port mini

Intrathecal Trial Update – Highs and Lows

Posted Saturday June 09, 2012 by Melissa

I’ve often said that a clinical trial is not for the faint...

I’ve often said that a clinical trial is not for the faint of heart and this past month should testify to that. We were at UNC from May 14-25. We...

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Bunny suit