Mom to Jordan, age 23, diagnosed in late 2005 at the age of 10. Jordan is graduating from Texas A&M in May 2018 and still able to walk medium distances but uses a scooter to get around campus. He has trouble with stairs and long distances but is doing extremely well otherwise. Genetic testing confirmed that he has Duchenne, but Steve Wilton and Dr. Wong told us they have found that boys with this particular mutation (deletion of exons 3-7) in general have much slower progression compared to boys with other mutations. He has been taking Deflazacort for 13 years. We started him at 30mg then reduced it to 22.5 mg when he had trouble sleeping and became self-conscious about having a fuller face. I think the combination of deflazacort, along with his being very thin (115 lbs) and having very good flexibility, has kept him on his feet for so long. Jordan will be starting a job in July 2018 with IBM in Dallas on their Cloud Development team. He will be living on his own in an apartment very close to his office.
About my family:
Married for 27 years to Stuart Taylor. He and I own a commercial lighting retrofit business. Son, Jordan (23), is first in family to be diagnosed with DMD. Daughter, Madison (20).
Name(s) of child(ren)/individual(s) with Duchenne:
Hey Donna, was thinking about you guys not to long ago. That is wonderful news that Jordan is doing well. I didn't realize he was a junior already. Will he be able to drive? We just got back from Dr Wongs last week, they said Jonluke was stable, but he hasn't grown in height at all in a year. They recomend him taking the growth hormone, and right now Dr Wong is doing study on them. She asked us to be in the study and every thing would be paid for. DId Jordan take them? Also we started the heart meds, they said he had a little fibrosis and we should start. He did well in the timed test they said he timed like a normal kid. THe PT said he was tight in the ham strings and finger flexors. We haven't been to good at the stretching but are starting back on it. How about Jordan does he do the stretching regularly? How about the night splints? Jonluke hates to wear them. I'm so happy that Jordan is doing so well, gives me more hope for Jonluke. Jonluke is doing well physically, he just started 6th grade at a new school. He has a rolling back pack this year which makes it so much easier, they would not let him have one in Elementary. Hope I didn't ask to many questions, Keep in touch
I think we just don't know if making more muscle is going to be enough in the long run...maybe, but my bet is that it will really work best to use something like ACE-031 to make more muscle and then a utrophin upregulator to stabiilze that new muscles. In combination these approaches might result in a really effective therapy. Unfortunately they will both have to be tested separately first, but if these drugs start coming on the market people will start using them in combination (as long as they are safe together). I don't know how recruitment is going in the Acceleron studies so far...I know they have two sites open now and are hoping to have a total of 6-8 eventually. We are talking to them about ways that we can help with recruitment and we may have an announcement to that end soon.
So, ACE-031 really is different from MYO-029 in that it blocks the entire pathway that myostatin was just one part of...it turns out that there is probably a whole group of molecules out there like myostatin that can put the brakes on muscle growth, If you block myostatin you get some effect, but you still have those other molecules out there doing their thing. But all of these molecules use the same cell receptor so if you block the receptor instead you take them all out with one fell swoop. That's what ACE-031 does. If you compare it directly in the mice to just blocking myostatin you get signficantly more muscle mass by blocking the receptor.
One encouraging thing is that in the trial of ACE-031 in healthy volunteers the volunteers saw a significant increase in lean muscle mass. It's not clear that this was the case with Wyeth's MYO-029 drug. I
Hi Donna! My husband has talked to Stuart. I guess they connected thru some GE affiliations...if I have the story right, your husband worked with Bill Hudson who is a good friend of ours. I am glad your son is doing so well- it gives us all hope!! Jonathon has a larger deletion of 8-31. He seems to be doing well, he has a great attitude and thinks that he has the ability to do everything like everyone else! We got into Cincy in early Aug. so he has been on the Deflazacort which we can see a difference in the energy level!! That is good. I think the hardest thing is trying to let him be a kid and deciding when enough is enough!! Curious to know what Jordan's deletion is- but then does it really matter? Would love to talk with you sometime. I will send you my email address.
Donna, good to hear from you, I love hearing that Jordan is doing so well, gives me hope. How did you find out that Jordan had DMD? We came across it by accident, my home page tells how. Jonluke is also very bright and loves to build things and scored very high on an IQ test that our public school gave him. He had problems with distractions and staying focused, that is what led us to this diagnosis. Does Jordan have an IEP? We just got ours in place for this school year. He will have modifications when needed. Were in small town called Magnolia and there is at least 10,000 kids in the district and Jonluke is the only kid they have had with DMD. I had to do a lot of educatiing on the diease. Thank goodness every one in his school has been very supportive and will do what ever we need done. Also is Jordan taking steroids, I can't remember if I read if he did or not. Jonluke has had a huge energy jump since being on them. If Jordan is on them, did you have any issues with gaining weight? Jonluke is the pickiest eater, it has to have the right texture and consistency or he won't eat it, I'm not to concerned that he will gain excessive weight, but will keep a close eye on it. Let's keep in touch
Donna, Hi, I haven't posted to you yet, haven't been on here much in along time, our sons have the same deletions and we also live in Texas and go to Dr Wong, so looks like we have alot in common. I'm a couple years behind you, my son is 9. It's great to see your son is doing so well, I hope mine is doing as well as yours in 5 years. Jonluke is doing great since he started the steroids, it's hard to beleive he as MD, he is always going, he keeps up good with his peers much better now. He does get tired on long distances but so does some of his peers with out MD. We should stay in touch and keep each other informed since we share the same deletions.