Blog

Uncontrollable

Posted Sunday June 01, 2014 by Melissa

Last week, I had a not so gentle reminder that this disease,...

Last week, I had a not so gentle reminder that this disease, and the medical trauma caused by attempting to treat it, still have a strong grip on my child....

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#PurpleFest aka MPS Awareness Day

Posted Wednesday May 14, 2014 by Melissa

Tomorrow is all about the purple! Happy MPS Awareness Day! We spend...

Tomorrow is all about the purple! Happy MPS Awareness Day! We spend the day sharing about MPS to educate and raise awareness, to make a better community for our children,...

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Cure

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

Death is My Neighbor

Posted Thursday March 20, 2014 by Melissa

* An essay inspired by the struggles of our MPS II community...

* An essay inspired by the struggles of our MPS II community and the loss of the boys we love so dearly Death is My Neighbor He comes a knockin’...

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New shirts have arrived!

Posted Wednesday March 12, 2014 by Melissa

Just in time for Case’s 7th birthday today, the new Saving Case...

Just in time for Case’s 7th birthday today, the new Saving Case & Friends T-shirts have arrived! Visit the gear page to order today and raise awareness for any rare...

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Rare disease awareness shirt

Dear child who will never be “normal”

Posted Wednesday January 29, 2014 by Melissa

Dear child who will never be “normal,” I love you. Let me...

Dear child who will never be “normal,” I love you. Let me say that first, because it is the most important. I love you as you are. As you grow...

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Case - a lesson for us

#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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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

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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Why don't we get wet?

OPEN LETTER to Dr. John Leong Chi-yan and the Hong Kong Hospital Authority re: Access to Idursulfase for the Tang brothers

Posted Tuesday November 26, 2013 by Melissa

**SIGN the Change.org petition to support the Tang brothers!** OPEN LETTER to...

**SIGN the Change.org petition to support the Tang brothers!** OPEN LETTER to Dr. John Leong Chi-yan and the Hong Kong Hospital Authority re: Access to Elaprase/Idursulfase for the Tang Brothers...

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Tangbrothers

Path to Diagnosis: Through an ENT?

Posted Saturday November 23, 2013 by Melissa

We all have different stories to tell about our child’s path to...

We all have different stories to tell about our child’s path to a diagnosis, whether it be for MPS or another condition. For rare diseases, often that path is long...

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Embracing Medical Care

Posted Friday November 08, 2013 by Melissa

If you haven’t seen this great video yet, you need to. If...

If you haven’t seen this great video yet, you need to. If only all doctors and nurses understood the emotional need for patients to “embrace” their care – something that...

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DrCase

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

Why I don’t unplug (and don’t feel guilty about it)

Posted Thursday September 19, 2013 by Melissa

As with any unhealthy trend, there is often a backlash, merited or...

As with any unhealthy trend, there is often a backlash, merited or otherwise. Smoking? Anti-smoking campaign. Obesity? Get healthy campaign. Alcoholism? Sin tax. Digital addiction? Soft and gentle suggestions to...

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Rare Disease Book Review: Brain on Fire

Posted Tuesday September 17, 2013 by Melissa

Brain on Fire is the extraordinary story of an ordinary woman. Susannah...

Brain on Fire is the extraordinary story of an ordinary woman. Susannah Cahalan was an ordinary woman who was in the midst of living her life as a New York...

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Brain on Fire cover

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

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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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