Getting ready for the normal high school milestones in our house is always a creative endeavor. At the beginning of April Jon came home from school and said that he wanted to attend Prom. He asked if he could attend the prom without renting a tux. Our goal is to have Jon experience all the normal things kids his age get to experience. I said, yes we should get a tux. The month of April flew by. On May 1, Jon and I went into to the local tux shop to get measured. Prom was just two weeks away. Finding cloths is always a little challenging, due to taking steroids for 11 years. Jon’s growth has been stunted; he’s the shortest guy in his junior class. Most of the girls are taller than he as well.
The jacket and vest had to be altered. The pants had to be shortened. The shirt sleeves were really long. The tailor and the owner of the shop were both taking measurements and discussing what to do. Since it was to be a rental, the tailor was trying to figure out how to alter everything without cutting it. As Jon and I stood there listening to their conversation, Jon asked how much more it would cost if we purchased the jacket and the pants of the tux. The shop owner looked at me and said are you sure? Jon said, hey I’m not going to grow anymore, and I will be going to my senior ball next year. The owner quoted me a rental price and then gave me the purchase price. I said we will purchase the jacket and the pants. I will rent the shirt and vest. The tailor looked at the vest and figured out a way to make the alteration so they could rent the vest again. The shirt sleeves were solved by making them French cuffs. The tux shop provided the shirt studs and the cuff links. OK, the tux issue was solved, now for the shoes.
Jon’s shoes are always difficult. Jon has custom made inserts in his shoes. He usually always wears sneakers, because the arch supports can be removed and his inserts can be slipped in easily. Dress shoes present a problem. The arch supports can’t be removed as they are glued in, so trying shoes on with his inserts is difficult. We tried on dress shoes, no luck. As I stood in the shoe store, I started looking at sneakers. I saw a pair of black suede sneakers that were all black. We tried them on; they fit, and were comfortable. We purchased the shoes.
I got home both my husband and daughter said those aren’t dress shoes. I said I know, but they are the closest thing we could find and with his tux they will be fine. They both looked at me like I was crazy. Well, sure enough, they looked fine with his tux. You really had to look at them to figure out they were sneakers. Jon was comfortable and able to walk.
Jon looked great in his tux and he enjoyed himself at the prom and glad that we got a tux. The girls noticed him and took their picture with him.
Wednesday, May 19, 2010
Friday, May 7, 2010
Partial Wind in My Sails
The wind in my sails may be coming back.
Two things have occurred that provide me with some hope that Ataluren may still be viable.
One was the conference call PPMD held with John Crowley. John Crowley is a dad with two children diagnosed with Pompe’s disease. His story was the inspiration for the movie “Extraordinary Measures” starring Harrison Ford, Brendan Frasier and Kerry Russell. John Crowley is also the CEO of a bio-tech firm. John made some very interesting points about drug development that hit home with me. Drug Trial planning is very difficult. If the drug does not meet their chosen end points, it does not always means that the drug is not viable, harmful or bad. It could be as simple as the dosage may not be correct.
The other thing that provided both my husband and I some hope was the Summary of Ataluren Phase 2b Clinical Trial Results presented at the American Academy of Neurology Meeting on April 16, 2010. These results indicated that the low dose of Ataluren was the dose that showed actual benefit in some of the trial participants.
Both of these things have caused me to sit back and think about some of the complexities of Duchenne. Hitting the expected targets the first time out in genetic disease trials are very difficult. This caused me to think that endpoints like the 6 minute walk test and other tests that are reliant on a multitude of factors may not be the best end points for Duchenne. For example: toe walking; vs flat feet; pelvis strength; thigh strength; spine strength; steroids or not. I’m not sure that you can use such an end point and still be able to calculate all the different variants, to come up with an average of how the kids are performing during this test and whether or not a benefit has been achieved. Then on top of these factors, let’s add that some kids may not like the taste of the drug. A parent’s goal while participating in these trials is to make sure that their child takes the drug. Therefore a parent may be mixing the drug with something that might cause the drug to become ineffective without realizing it. All of these factors make analysis extremely complicated.
It’s not surprising to me the analysis has taken a little time. I believe that PTC as a company has not given up on the drug and that they are still regrouping to figure out the next steps forward. I hope PTC can determine the next steps soon. We are hoping that the trail will be resumed using the low dose and possibly different targets for analysis.
Therefore the wind is slowly coming back. The waiting game is always difficult.
Two things have occurred that provide me with some hope that Ataluren may still be viable.
One was the conference call PPMD held with John Crowley. John Crowley is a dad with two children diagnosed with Pompe’s disease. His story was the inspiration for the movie “Extraordinary Measures” starring Harrison Ford, Brendan Frasier and Kerry Russell. John Crowley is also the CEO of a bio-tech firm. John made some very interesting points about drug development that hit home with me. Drug Trial planning is very difficult. If the drug does not meet their chosen end points, it does not always means that the drug is not viable, harmful or bad. It could be as simple as the dosage may not be correct.
The other thing that provided both my husband and I some hope was the Summary of Ataluren Phase 2b Clinical Trial Results presented at the American Academy of Neurology Meeting on April 16, 2010. These results indicated that the low dose of Ataluren was the dose that showed actual benefit in some of the trial participants.
Both of these things have caused me to sit back and think about some of the complexities of Duchenne. Hitting the expected targets the first time out in genetic disease trials are very difficult. This caused me to think that endpoints like the 6 minute walk test and other tests that are reliant on a multitude of factors may not be the best end points for Duchenne. For example: toe walking; vs flat feet; pelvis strength; thigh strength; spine strength; steroids or not. I’m not sure that you can use such an end point and still be able to calculate all the different variants, to come up with an average of how the kids are performing during this test and whether or not a benefit has been achieved. Then on top of these factors, let’s add that some kids may not like the taste of the drug. A parent’s goal while participating in these trials is to make sure that their child takes the drug. Therefore a parent may be mixing the drug with something that might cause the drug to become ineffective without realizing it. All of these factors make analysis extremely complicated.
It’s not surprising to me the analysis has taken a little time. I believe that PTC as a company has not given up on the drug and that they are still regrouping to figure out the next steps forward. I hope PTC can determine the next steps soon. We are hoping that the trail will be resumed using the low dose and possibly different targets for analysis.
Therefore the wind is slowly coming back. The waiting game is always difficult.
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