Clinical trial results are in!

October 16th, 2016

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On Friday, Clementia Pharmaceuticals sent out an exciting press release. This was MUCH anticipated in the fibrodysplasia ossificans progressiva community. The subject of the release was, TA DA, the results of the first ever post-gene-discovery-era clinical test of a drug for FOP! So here's the scoop... In 2014, Clementia  started a Phase 2 clinical test of palovarotene, which I will call "PV" for short (they were able to skip over Phase 1 - testing for toxicity using unaffected patients - because another pharma company had done Phase 1 while considering PV for a different disease). In simplest terms, PV has been ...

The reality of doctors and rare diseases

October 9th, 2016

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Doctors... We all need them, but when you're coping with a rare disease such as my daughter's, fibrodysplasia ossificans progressiva, the experience can be by turns incredibly great and extremely frustrating. Some doctors are rock solid and terrific, while others make you want to rip your hair out at the roots. There are no doctors in this photo, but... This was Miranda in San Francisco, where she was meeting with a really top notch physician. Let's start in the pre-diagnosis phase. So, you have a child, and the child has some strange, seemingly inexplicable medical symptoms. Chances are your child will be ...

The early days of an FOP diagnosis

October 2nd, 2016

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There's recently been a cluster of newly diagnosed people who've joined the FOP community; I learn of this when each has a family member added to a private FOP Facebook group to which I belong. Every time I see one of these newbies, which are almost always children, I'm taken back to when my family were in this situation, way back in 2007. Saying that this is a "hard time" is putting it in the mildest possible terms. There's ever so much to figure out - which, of course, got me thinking that this would be a good topic for ...

The genetics of FOP

September 17th, 2016

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I was recently in a bookstore and looking to spend a gift card when I stumbled on an interesting item, which I bought. Science nerd that I am, I've been finding this book absolutely fascinating. It's called "The Gene: An Intimate History", and it's by Siddhartha Mukherjee (no, I am not getting paid to say this, LOL). The book is all about the history of discoveries connected to the gene. One paragraph so far particularly stood out to me. In discussing the disease sickle cell anemia, Dr. Mukherjee had this to say: "It was a Rube Goldberg disease. A change in ...

Back to school, 2016 edition

September 11th, 2016

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Here we are in late summer, and it's "The MOST WONDERFUL TIME of the YEAR!" (to quote that ubiquitous September TV commercial, LOL). We did the lazy summer days thing, enjoyed a long Labour Day weekend, and then it was finally time for school. This is good, as far as I'm concerned, since my kids were definitely ready to go back. [caption id="attachment_3008" align="alignleft" width="600"] Summer fun (obligatory frappuccino in hand).[/caption] My daughter Miranda is 11 and started grade 6 this year. Though she won't admit it - or at least will qualify her answer in various different ways - my girl ...

Philosophy and FOP

August 21st, 2016

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A few weeks ago, someone made a strongly worded comment on one of my blog posts. In my post, I had mentioned about how much I hated fibrodysplasia ossificans progressiva. My commenter called me out on this, stating that she wondered how my daughter Miranda(now age 11) would adapt as she grows, knowing that I hate something which is a part of her. Ouch. I have to admit, this kind of knocked the wind out of me. And yet, the comment did get me thinking... Since then, I've been sort of turning this issue over and over in my mind at random ...

Summer challenges

August 14th, 2016

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Just a few days ago I was chatting with one of my neighbours. We got to talking about what our kids are doing this summer, and she told me about a summer day camp which her kids had attended. She thought this camp was great, and that I should consider it for Miranda next year. I didn't say much - just smiled politely. You see, getting summer camps sorted out for kids like my daughter, who has a disability, requires some effort. Because of Miranda's fibrodysplasia ossificans progressiva, I can't just register her for any old thing and then have her ...

Summer vacation, and Walk for FOP 2016

August 4th, 2016

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It's Thursday, and we've been back from our summer vacation road trip for a couple of days. Here's what we did on our epic journey (to quote my son, ha ha)... Last Sunday we got in our minivan and drove to Kamloops, BC for an overnight stop, then carried on the next day to Invermere. At Invermere, we met up with my parents, my siblings, and my nieces and nephews for a joint vacation. So. Much. Fun! We were in Invermere together for 5 days, and did all the great stuff that summer vacations are for. We swam in Lake Windermere, had ...

I suck at fundraising, or, 2 weeks till our event!

July 17th, 2016

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I'm going to admit this right here and now - fundraising is not my strong suit. Ask me to write some stuff? Sure, I'm your gal. Need me to do a verbal presentation? You bet, no worries. But fundraising... Oy. In this particular arena, I lack creativity, confidence, and general all around know-how. And yet, fundraise I must. Why do I gotta do this stuff, at which I have crappy skills? Well, let's go back in time 9 years, to 2007 when my daughter Miranda was a spunky, cute, and sassy 2 year old. On April 19 of that year, the ...

FOP in San Francisco

July 9th, 2016

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During the last week of June, my daughter Miranda and I took a trip to San Francisco in connection with Clementia Pharmaceuticals's ongoing work with fibrodysplasia ossificans progressiva. I won't go into specifics about the reason for our visit, but will simply say that Miranda was doing her part to move FOP research forward. It was a short trip; we just flew in on the Wednesday evening, spent the night and next full day there, and then flew home Friday morning. Still, what a place to be. I love San Francisco, which is very scenic and interesting. It's also close to ...
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