Monday, April 22, 2013

Senior Project and Science Project Focusing On Mito

My sweet niece has made a wonderful Mito awareness video, but for some reason, I cannot get it into a form to share. Sad, but I will figure it out. My niece is 12, and the video is FANTASTIC!! She shared it with her class and then again with another organization. I am so very humbled that she chose to focus on Mito at the local level and raise awareness. I love this precious little gal so much, and I know from the care she shares with her cousins and the artistic talent and attention to heart and detail she used on the video, that she is going serious places, and will help others in HUGE ways along her journey!! I am so so so proud of her!!!

AND THEN.......

A senior student at our local, small town USA high school approached me about doing her senior project on Mitochondrial Disease Awareness. First, I was speechless! I know this amazing young lady and her fantastic family from our involvement in 4-H, and I already liked her a lot, but this pushed her into the LOVE category! She has met with me twice, shared her ideas, taken over bracelet-making, written talks, scheduled presentation to multiple high school and middle school classes, and is even doing an awareness video!! SO tonight, while photos are being uploaded to her for the video, I had to get over here to share this fantastic news!!!!

Every bit of awareness is KEY to SOLVING the problem of Mitochondrial Disease. Never discount the value of even just telling one person about Mito!!!!!


Monday, April 8, 2013

My Boy Further Amazes Me!!

I have to post a quick report on a wonderful evening we had Saturday with very dear friends. We arrived at their home around 4 and stayed for a hotdog cookout, wonderful homemade salsa with chips and enough fixins and snacks to sink a ship!!!

But even though the company was FABULOUS, the food was DELICIOUS, and the evening was WONDERFULLY FUN, that's not what stands out.....well, it does, but one other thing beats it! Garrett was with us the whole time, and he was such a great boy!!! As recently as six months ago, this long of an evening or time out in one location would have flopped. Actually, about two and a half to three years ago, we completely gave up taking Garrett with us on those sort of outings. We would always get a sitter or just not go. More often, we wouldn't go. Friends (not all, but many) in a former town, just plain stopped inviting us to things since they knew we typically wouldn't come. We began to feel a little isolated and left out, honestly. No, this wasn't our very closest friends, but it was those fringe friends and large groups of friends and acquaintances. We did have SUPER love and support from our church, family and friends back then, so don't anyone take this wrong!!! But it was a feeling we had never felt as a couple. Isolated was the last advective you would have used when describing us!! I tell you this to paint the picture of how amazed and THRILLED we were to be leaving a family's home near eleven at night with a HAPPY BOY who between friends, iPad, my phone and his sisters, was completely calm, happy and entertained.

I am still beaming!! I have so much more greatness of Garrett's to share, but I need to get some sleep. I hope to share more and more of the GOODNESS going on with our little hero. My deepest desire is to help you see very clearly how God is at work and how YOUR PRAYERS are yielding huge progress and change in our son and our family. I love each of you and cannot thank you enough!!


Tuesday, April 2, 2013


I cut and pasted this from another mom's blog, and I know this can mean HUGE things for our child and thousands of others in the maybe not too far away future if things like this keep happening!!!!

EPI 743 gets $545 million from Japan's Dainippon Sumitomo

This is a HUGE win!! If you happened to watch that TED Talk I posted a few days ago, it talks about how many years it takes to get a drug from testing to market (14 years) and how much money (billions). 

Edison Pharma Inks potential $545M Deal with Japan’s Dainippon
March 29, 2013 
From BioWorld: ”Privately held Edison Pharmaceuticals Inc. wooed a big pharma partner, inking an R&D and commercialization agreement with Dainippon Sumitomo Pharma Co. Ltd. (DSP) to develop lead program EPI-743 and follow-on molecule EPI-589 in Japan.

Terms called for Edison to receive $35 million up front and $15 million in R&D support. The Mountain View, Calif.-based biotech also will be eligible to receive $10 million to $35 million in development milestones per indication and up to $460 million in commercial milestone payments, as well as royalties on commercial sales. DSP gained development and commercialization rights to the compounds in Japan, but no ownership or control over development activities elsewhere.

EPI-743 is an orally bioavailable small molecule in development for inherited mitochondrial diseases. A member of the para-benzoquinone class of drugs, the compound targets the enzyme NADPH quinone oxidoreductase 1. EPI-743 is in U.S. Phase IIb trials in Leigh syndrome and in Friedreich’s ataxia, both ultra-rare indications.

The initial scope of the transaction includes both pediatric orphan inherited mitochondrial and adult central nervous system diseases. DSP will assume activities required for development, approval and commercialization of EPI-743 in Japan, initially focusing on orphan pediatric mitochondrial disease.”

And another story on it here, including an interview from Guy Miller, Edison Pharma's CEO.