Morquio Syndrome: How One Inspirational Man Refused to Take “No” for an Answer

I have a new hero, an Irishman who’s not only gotten some great press and notoriety, this guy has struck gold in the hearts of the Morquio syndrome community!

In a strange twist of fate the treasure this gentleman , who lives with Morquio syndrome, a rare genetic and progressive disease that prohibits growth and the development of bones, discovered is something far more valuable than gold. It’s a life-changing drug (at least for him–remember that everyone responds differently to treatment!) called: Vimizim.

An infusion treatment that’s administered in an infusion center and is widely available to patients in Europe, Vimizim wasn’t available to people in Ireland. Without access to this treatment, his health worsened as his disease progressed. He had little stamina and was dependent upon assistive devices, including an electric wheelchair and ventilation machine, to keep his airway passages open when he slept at night.

So after “having enough of not getting any,” he methodically began his own fierce awareness campaign. He wrote letters to the heads of government in the various political parties. He outlined the tragedy of how this rare disease affects people, with numerous hospital stays, needing intensive care treatment, and how this can potentially be avoided if patients could receive Vimizim.

And he was granted his wish.

I’m not sure exactly how long it took, but the pharmaceutical company that makes Vimizim announced the drug would be made available in Ireland!

And the rest, as they say, is history! Although he felt victorious, he knew that there were a handful of other Irish patients in dire need of the drug who didn’t have access like he did, so he called on their behalf and I’m guessing he didn’t mince words when it came to communicating how desperate they were for the treatment.

I don’t know much about the healthcare system in Ireland, but I’m wondering if the government paid for the drug? It would probably cost a fortune here in the States!

Alisha Stone

Alisha Stone

Alisha Stone has a BA in psychology and is dedicated to improving the lives of others living with chronic illnesses.

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