Posts tagged with shire-hgt

Happy birthday Elaprase!

Posted Wednesday July 24, 2013 by Melissa

Today is the 7th anniversary of the FDA approval of Elaprase (idursulfase)...

Today is the 7th anniversary of the FDA approval of Elaprase (idursulfase) as the first treatment for Hunter Syndrome. On July 24, 2006, the FDA approved Elaprase. It provided HOPE....

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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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The Systemic Impact of Rare Disease: Medical, Emotional, Financial, Social

Posted Tuesday April 09, 2013 by Melissa

Rare diseases may seem like solely a health care issue, but in...

Rare diseases may seem like solely a health care issue, but in fact, a newly released report finds that their impact is much more systemic, affecting the health care system...

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US rare disease infographic

Shire HGT’s #RareDisease Developments

Posted Saturday March 23, 2013 by Melissa

For those of us intimately involved in the future of Shire and...

For those of us intimately involved in the future of Shire and its division Shire HGT, either because we or our kids already use a Shire product (us), because we’re...

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Go Ahead and Stare

Posted Thursday January 24, 2013 by Melissa

I recently wrote an article that was published today in the Positive...

I recently wrote an article that was published today in the Positive Exposure newsletter about my experience visiting the Hunter in Focus exhibit this year in NYC.  Go Ahead and...

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Live at “Hunter in Focus”

Posted Tuesday October 30, 2012 by Melissa

Success. The event was a success. Lives were touched, awareness was raised,...

Success. The event was a success. Lives were touched, awareness was raised, and connections were made. Were Case there, he would have pointed at the tall picture of himself, danced...

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Hunter in Focus

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

What we want from (big) pharma

Posted Monday April 23, 2012 by Melissa

During my recent time at the World Orphan Drug Congress, I had...

During my recent time at the World Orphan Drug Congress, I had the pleasure of a nice dinner and conversation with some operations folks from a larger pharmaceutical company, otherwise...

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Case enjoyed the World Orphan Drug Congress (& so did I)

Posted Thursday April 19, 2012 by Melissa

Last week, I was incredibly blessed to attend and speak at the...

Last week, I was incredibly blessed to attend and speak at the World Orphan Drug Congress. Why did I go? First, because of course I’ll take advantage of any opportunity...

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Reversal of Fortune? Where Profits and Patients Collide

Posted Monday April 09, 2012 by Melissa

The rare disease world, and especially the non-precisely-defined ultra rare disease world,...

The rare disease world, and especially the non-precisely-defined ultra rare disease world, is a small one. You know the players. You meet over and again. Your pharma company knows your...

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What I wish I’d known about home infusions

Posted Wednesday November 30, 2011 by Melissa

Setting aside a day of your life each week to go to...

Setting aside a day of your life each week to go to the hospital can be hard. That alone is hard, but add to that the lunch and supply packing...

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Elaprase

Anatomy of an Intrathecal Clinical Trial Visit

Posted Friday October 28, 2011 by Melissa

Now that we only have 4-day visits instead of 9-day visits, things...

Now that we only have 4-day visits instead of 9-day visits, things seem to go much more quickly. There’s definitely time for some fun stuff (less now that the trips...

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MPS II Intrathecal Trial on Hold

Posted Wednesday June 15, 2011 by Melissa

UPDATE: As of 7/1/11, the suspension on enrollment in the intrathecal trial...

UPDATE: As of 7/1/11, the suspension on enrollment in the intrathecal trial was lifted by the UNC Institutional Review Board. ———————————————– A clinical trial is never a place to get...

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Taking Time

Be Still My Beating Heart

Posted Friday April 29, 2011 by Melissa

(Humming to Sting as I type this) I really did have to...

(Humming to Sting as I type this) I really did have to still my heart when I heard the latest news. So as not to steal the Thomas family’s thunder,...

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Heart anatomy

Top 10 Tuesday: Top 10 Signs Your Son is in a Clinical Trial

Posted Tuesday April 19, 2011 by Melissa

Welcome to our first Top Ten Tuesday! On a side note, if...

Welcome to our first Top Ten Tuesday! On a side note, if you have any submissions or ideas to use as future Top 10 Lists, feel free to submit them...

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Top 10