The FOP one was the BEST table at the rare disease conference…

Posted by Karen - April 2nd, 2017

On Thursday and Friday of this week, I attended the annual conference of the Canadian Organization for Rare Diseases (CORD). I’m used to being the only person in the FOP community at these things, but not this time… On this occasion, I and two other moms of kids with fibrodysplasia ossificans progressiva attended as guests of Clementia Pharmaceuticals. Clementia is the company doing clinical testing of Palovarotene, a potential drug to treat FOP, and it sent along two representatives – Stephanie Hoffman and Eric Grinstead. The result was that there were 5 OF US sitting together at the conference. We had a full-on FOP contingent! Definitely a novel experience with FOP, ha ha. The two other FOP moms I was with were Carrie Connell, president of the Canadian FOP Network and mother of a teen girl with FOP, and Stephenie (“Steph”) H (not to be confused with Stephanie Hoffman of Clementia), also mother of a teen girl with FOP. Steph H and I both live in the local area, and Carrie flew in from London, Ontario.

Steph, Carrie, and me

Steph, Carrie, and me

The conference was about all things connected to rare diseases. As such, there were research presentations, patient panels, workshops on rare disease topics, and more. “Team FOP” actually contributed two, count them, TWO, bits to the conference – both Carrie and Stephanie were in the spotlight.

Carrie was on a patient panel. She had a 15 minute window to talk about what it was like to get a rare disease diagnosis for her daughter, and the challenges in finding useful and appropriate information and medical help. Carrie spoke eloquently and with feeling, and it was my sense that other participants at the conference found her words to be moving and compelling. Here’s a pic of Carrie doing her thing:


As for Stephanie, she was on a panel of research presenters. The other individuals on this panel each spoke about the fine details of various aspects of rare disease research in which they are involved, and while it was mildly interesting, these were overly technical presentations with little or no relevance to the broader context of the conference. Stephanie, on the other hand, talked about Clementia’s narrative project, in which they conducted interviews of patients and physicians in the FOP context. She explained that an important focus for them was in trying to understand the factors which would be involved in an FOP patient being able to access a treatment (when it eventually becomes available). These factors, as it turns out, are variable from region to region, country to country. This presentation, in contrast to the others on the panel, was of interest to a broad range of people, as the principles involved matters which would be relevant to treatment of any rare disease. There was a lot of interest in Stephanie’s commentary; a number of people told me afterward that they found her subject to be interesting and useful to them.


The dynamic Stephanie Hoffman!

The dynamic Stephanie Hoffman!

In conjunction with the conference, there was also a gala dinner at which awards were presented to various persons working in the rare disease context. The gala was super glamorous and fun! Of note, entertainment was provided by a young man, 17 year old Justin LaBrash, who is a country music performer and who himself has a rare disorder. Justin was extremely talented, and his music was very enjoyable. I was especially amused to note that Stephanie from Clementia, who lives in Belgium, is a big country music fan! WHO says country music only appeals to Canadians and Americans? ;-)

Many thanks to Clementia Pharmaceuticals for inviting Carrie, Steph H, and me to attend the CORD conference. Additionally, I will add that Stephanie and Eric were exceptionally friendly and gracious, and I felt great rapport with them. What a nice couple of folks! I am invigorated and very much looking forward to Clementia’s attendance at our June 2017 Canadian FOP Network family conference.

Me, Stephanie, Eric, Steph, and Carrie

Me, Stephanie, Eric, Steph, and Carrie (Eric used a selfie stick! unfortunately photo is a bit blurry)


All in all, the conference was terrific, and I am grateful to have attended.

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