An Ordinary Day
On May 15th, International MPS Awareness Day was observed. For most of the world, this was not a grand event. From what I could tell, it was business as usual — just an ordinary day. But “ordinary” is...
View ArticleNew Resources That Will Help Speed Diagnosis of Rare Diseases
The rare community has made huge strides in the 30 years that have elapsed since the Orphan Drug Act was passed. There are more new treatments and there is more investment in research and more...
View ArticleMothering A Child With Special Needs
When I was pregnant, I was told that my son would be born with holoprosencephaly – a very serious condition that most children don’t survive. In fact, so few children survive that I really didn’t know...
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Cystinosis Community Celebrates Rare Disease Day through Med-Art
Over the last couple of weeks, our community of cystinosis families has been enriched by an exchange of “med-art” that began with a Facebook post by Clinton Moore, a dad of a child with cystinosis....
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Trivia can Save Lives: How a Random Jeopardy Question Can Raise Awareness of...
I woke up this morning in a great mood. Something wonderful happened last night. Let’s see, what was it? Oh yeah, that Jeopardy question. Ugh. Then there was the realization that the moment was...
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Saving Eliza
I’m honored to be speaking at the NORD Breakthrough Summit on Rare Diseases and Orphan Products in October. Social media, as I continue to learn more every day, is an always changing, rapidly...
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