![an·tic·i·pa·tion [an-tis-uh-pey-shuhn] noun realization in advance; foretaste. intuition, foreknowledge, or prescience. I met Julie Reneer in passing once, at the second National MPS Society conference we attended. And as with...](http://www.savingcase.com/wp-content/uploads/2012/05/life-and-death-in-kites-195x110.jpg "Anticipation … and flying")
![birth·day [burth-dey] noun the anniversary of a birth. the day of a person’s birth. a day marking or commemorating the origin, founding, or beginning of something. the festivities or celebration marking...](http://www.savingcase.com/wp-content/uploads/2012/03/Case-5bday-195x110.jpg "What’s in a [burth-dey] anyway?")
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My (un)Planned Son- I've never been a great writer May 14, 2012I've never been a great writer, a creative writer at least. Oh, I liked legal writing. It was structured and formulaic. But the writing I've been doing this past year is anything but. It is raw. It is painful at times. When Case was diagnosed, and then when he was to enter the clinical trial, I felt like God set tasks before me of creating Case […]
- Raise your hand if you want to be an American martyr? April 26, 2012I was watching a TV show the other night where a mom had been kidnapped. The kidnappers wanted information from her and they were torturing her to get it. It struck in my mind the analogy to Christians who are tortured for their faith either in the past, like in the Bible, or presently in some countries. The torturers generally want them to renounce their fa […]
- I've never been a great writer May 14, 2012
Featured Stories
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Anticipation … and flying
an·tic·i·pa·tion [an-tis-uh-pey-shuhn] noun realization in advance; foretaste. intuition, foreknowledge, or prescience. I met Julie Reneer in passing once, at the second National MPS Society conference we attended. And as with…
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MPS/ML Bookshelf
You may have noticed that rare disease finds its way into books, and on television and movies here and there. There’s nothing more popular for rare disease geeks than House,…
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Monitoring Ultra Rare (MPS/ML) Pharma
Prior to MPS coming into our lives, my job was as a strategic consultant, mostly to law firms. I sometimes joke that MPS still requires that I be a strategic…
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Sometimes we run, when we don’t want to
Today I have a race. A race I didn’t train for. And I’m running alone. Will I finish? I hope so. Will I collapse? I hope not. Will I do…
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Can I run too?
We live at the end of a long gravel lane, about one third of a mile long, back in the woods. It is not uncommon for my older boys to…
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What we want from (big) pharma
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)
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
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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Doing our Part in the Rare Disease World
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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Ebook Calmer is now available!
Today is the early release date of my ebook, Calmer: Medical Events with Cognitively Impaired Children, on Smashwords and Amazon Kindle! On Smashwords, you can preview 20% of the book…
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Upcoming e-book release!
I am so excited about next week’s release of my e-book Calmer: Medical Events with Cognitively Impaired Children. This e-book started out as a response to an e-mail, then became…
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What’s in a [burth-dey] anyway?
birth·day [burth-dey] noun the anniversary of a birth. the day of a person’s birth. a day marking or commemorating the origin, founding, or beginning of something. the festivities or celebration marking…
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Hope … and Chaos: Rare Disease Day
Today is a unique day. It is known to most as being unique for being Leap Day. But for our family and those of many we know, it is unique…
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As we watch the bridge…
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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And his name was Flower…
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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Newly Diagnosed Families: Airport Advice
Many children with MPS or other special needs sometimes appear to others as just a “difficult child” instead of a child with a disability that causes certain behaviors and symptoms….
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Special needs means … special gifts
I loved being pregnant. Even though Case was a surprise, I just couldn’t get over the kicking, rolling, and hiccupping that delighted me each day. When he was born, he…
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Intrathecal Trial Update – Of Ports and Prayers
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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The Cure Theorem: a rare disease proposition
This post is part of a blog hop to promote World Rare Disease Day on February 29, 2012. Please visit the posts of the other participants listed at the end…
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This is for all the Calebs
Have you realized that not all children with Hunter Syndrome can receive treatment? In some countries, if their brain has been affected by the disease or when that effect is…
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Making a UNC Visit?
We’ve had a lot of interest from families about what to do, where to stay, how to get around, etc. when they make trips to UNC or Duke for MPS…
