Why an FOP disease registry?

Posted by Karen - July 12th, 2015

Are you familiar with the expression, “an embarrassment of riches”? In case not, that’s a situation where you’ve got lots and lots of a very good thing. That’s sort of the situation we’re in right now with developments toward treatment for fibrodysplasia ossificans progressiva. After my daughter Miranda was diagnosed with FOP in 2007 at age 2, we had a lot of years were it was a research article here, another one there, but nothing that felt really concrete… Until a couple of years ago. Since then, it feels like things have been exploding around us. Which is absolutely fab, I am NOT complaining! In recent times I’ve been able to post about the Palovarotene drug trial, the FOP Natural History Study, and more.

Which brings me to the newest thing coming up – an FOP registry. Pretty soon, the International FOP Association will be launching this project. The registry will be a special website at which a person with FOP, or their parent or caregiver, creates a profile of the FOP person which includes particulars as to identity, disease progression, degree of disability, symptoms, disease progression, and other related matters. Basically, once you enter the registry, it takes you through a series of questionnaires where you check off answers to a bunch of different questions. (I know generally what it looks like as I have been one of a number of folks involved in some test runs.) Once you’ve created your registry profile and entered the necessary information, you’ll be invited to update that information at regular time intervals, probably a couple of times per year.

So… What’s the point of all this? As I understand it, the value of a registry is that it will be able to “de-identify” pertinent and valuable disease progression information from personal details and provide that information (i.e., the disease stuff) to FOP researchers. This kind of data will be invaluable to scientists trying to figure out whether, say, a given drug or other treatment method has any noticeable effect on the ongoing progression of FOP. The registry represents an important piece of the puzzle, a way to track the real world effects of FOP on people’s day to day lives. The IFOPA’s registry follows in the footsteps of efforts by other disease groups, including muscular dystrophy and metabolic disorders.

The bottom line is that the FOP registry will be another way that folks with FOP can participate in helping research progress. All good! To read the IFOPA’s recent announcement about the registry, go to: http://www.ifopa.org/news-and-events/latest-news1/532-fop-connection-registry-a-step-in-our-journey.html

I believe the registry will be up and running and ready for use within the next couple of months.

In other news, please don’t forget about our upcoming FOP fundraiser. On August 2, 2015, our family and 2 other FOP families will be doing the “Walk for FOP” in Calgary (in honour of Miranda and Kathleen) and Vancouver (in honour of Alex). All funds raised will go to the Canadian FOP Network, a registered charity which financially supports FOP research. Research which will benefit from, oh wait, I know – the IFOPA’s registry! ;-) If you’d like to support our efforts, please donate at www.walkforfop.com and click on either “General Donations” or “Donate to Participant” (to select a specific person in respect of whom to donate). Or, contact me and I’ll tell you how to send a cheque – this route may be better for larger donations to save on credit card charges (levied by our fundraising website). If we can top $20,000 in donations this year, Miranda’s dad Pete and brother Owen will each do 100 burpees! That’s some difficult stuff, which I would love to see, ha ha. Help us reach that goal!

We walk for Miranda. (Don't you love this Carmen Miranda minion Tee? I thought is was perfect for Miss M.  :) )

We walk for Miranda. (Don’t you love this Carmen Miranda minion Tee? I thought is was perfect for Miss M. :) )



1. If you have FOP, are age 15 or over and have a new flare-up on an arm, leg, hip or shoulder, you may qualify to take part in the current trial for the potential FOP medication Palovarotene. It involves some short-term travel to one of 3 test sites in Paris, France, San Francisco or Philadelphia, but all travel expenses are paid, even if you have special travel needs. For more information, go to: http://www.ifopa.org/drug-development/clementia-palovarotene-clinical-trial.html

2. People with FOP who are age 2 to 65 may qualify to take part in the FOP Natural History Study. Ditto the comments above re: travel, though there are more available test sites for this project. For more information, see: http://www.ifopa.org/drug-development/clementia-fop-natural-history-study.html

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