Posts tagged with 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

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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DeclanMitchell-14

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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Melissa-Easton

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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Case sleeping

Is there a formula of risk tolerance to treat rare diseases?

Posted Monday January 06, 2014 by Melissa

What risk would you take with your child’s life? What if the...

What risk would you take with your child’s life? What if the treatment had a 25% chance of death or serious injury, would you risk it? The willingness of patients...

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Formula for assessing risk tolerance in rare disease

My New Year’s Resolution is…

Posted Saturday January 04, 2014 by Melissa

New year’s resolutions are a staple of our society. We resolve to...

New year’s resolutions are a staple of our society. We resolve to lose weight. To get organized. To spend less. To be unplugged. I resolve to save lives. Now, I...

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Case-Christmas

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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AIM-IT

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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TaleofTwoFrogs

Important new data and disclosures re: Shire’s Elaprase

Posted Friday July 19, 2013 by Melissa

Most of us probably don’t read that folded, fine-print enclosure that comes...

Most of us probably don’t read that folded, fine-print enclosure that comes stuffed in boxes of medicine, even if the medicine is Elaprase, one we give to our children for...

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Elaprase

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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jeep

Birthdays, Blessings, & Burdens

Posted Tuesday March 12, 2013 by Melissa

Today is Case’s 6th birthday and I could not let the day...

Today is Case’s 6th birthday and I could not let the day pass without acknowledging what a blessing he is in our life. I also want to note that not...

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This was his response to me telling him to "Take a bite!" I meant his SLICE, but he decided the whole cake looked better!

Intrathecal Trial Update – Words on a Page

Posted Monday February 18, 2013 by Melissa

It is quite strange to see the last few years of your...

It is quite strange to see the last few years of your life boiled down to a few paragraphs of text and a line on a chart. It is even...

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From Shire's Q4 2012 earnings call presentation

Letting Go

Posted Thursday July 05, 2012 by Melissa

We are in an interesting quandary, but a good one to have...

We are in an interesting quandary, but a good one to have I guess. We have many pieces of equipment and supplies that we acquired before the clinical trial and...

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Case in stroller

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

As we watch the bridge…

Posted Monday February 13, 2012 by Melissa

Clinical trials are not for the faint of heart. I think I...

Clinical trials are not for the faint of heart. I think I may have said that before, but I think generally because I am an upbeat and optimistic person, people...

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Old wooden bridge

And his name was Flower…

Posted Thursday February 09, 2012 by Melissa

Santa brought Case a pink-nosed Zhu Zhu pet for Christmas. Case runs...

Santa brought Case a pink-nosed Zhu Zhu pet for Christmas. Case runs like lightening, pretending that the hamster is after him when he activates it and the crazy thing starts...

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Brain maze

Intrathecal Trial Update – Of Ports and Prayers

Posted Wednesday February 01, 2012 by Melissa

We are in North Carolina on the tail end of our visit...

We are in North Carolina on the tail end of our visit for IT dose #15. The dose was successful, meaning they were able to access the port, draw off...

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Intrathecal tattoo