Posts tagged with shire

Rare Disease Clinical Trial Recruitment – What’s the Holdup?

Posted Monday July 28, 2014 by Melissa

For some parents, as soon as their child is diagnosed with a...

For some parents, as soon as their child is diagnosed with a rare, progressive disease, the first thing they do is research whether there is a cure, a treatment, or...

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AbbVie and Shire – A Rare Match?

Posted Saturday July 19, 2014 by Melissa

Ever since AbbVie’s initial approach to Shire became public, our Hunter Syndrome...

Ever since AbbVie’s initial approach to Shire became public, our Hunter Syndrome community has been watching and waiting. We’ve always known Shire was a candidate for an acquisition, although its...

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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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#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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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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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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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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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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Doing our Part in the Rare Disease World

Posted Friday April 06, 2012 by Melissa

On the heels of Rare Disease Day and Rare Disease Patient Advocacy...

On the heels of Rare Disease Day and Rare Disease Patient Advocacy Day at the FDA, NIH, etc. (where several of our friends participated – thank you!), this week is...

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