Just thinking about you and your husband and wondering how he is doing. My brother-in-law, age 57, was diagnosed with colon cancer, stage 3. He had surgery, then chemo for about 4 months, I think. He is doing well. He is hear this weekend from Mt. If there is anything you'd like me to ask him, let me know. He has his care here and in Mt.
I have a question - I am attending the Feb 4th Uni. of WA event for DMD put on by Leffler/Schneider families. Presenting will be Dr. Susan Apkon, Dr. Chamberlain, a guy from AVI and one other researcher on Losartan. Question for you....is there anything that you can suggest that I ask about it there is discussions?
I can't imagine how difficult it is to deal with your husband's diagnosis - on second thought - I can with the DMD. I am sending positive and prayers your way.
hi pat, i wonder what you know about the trial in columbus scheduled to begin in march. we have been asked for dna and steroid info on our two sons. do you know if boys who enroll in phase 1 study get to then move to phase 2? have you heard anything about prosensa planning trials in the US? i met the prosensa rep in london and he said early 2010. i worry about my oldest son not meeting trial criteria, i dont think he will be walking much longer. thanks for any info you have, jenn
I hope that something positive comes of Dr. Riordan's work. We have met Ryan and think this is just wonderful for him, he really does appear to be doing much better than even when we saw him at the telethon. This may not be a cure, but it would sure be great if we could get these treatments available here in the US. Our son and his wife stored the cord blood from their 4th child and are hoping to find a way to get stem cells grown and see if they will be a match for their son, Dominic. If this can even just help the DMD young men to extend their life until the Exon Skipping or some other 'cure' does come along then everything that can be done, to speed up this process making it available for all, should be done now not tomorrow! Cancer and Heart Patients are benefiting from Adult Stem Cells, even if they don't completely stop the Cancer, they are prolonging the patient's life and isn't that the goal here as well?
Just wondering about Joshua. Why did he go so soon? Also wondering if you have information about how Duchenne affects girls? One of our granddaughters (Dominics sister) seems to have quite a pronounced pigeon toe walk and I think it may be related to the Duchenne, her mother says that she had the same problem when she was little, so just checking.
Pat, I know you are a busy busy woman, I just wanted to tell you thank you for responding to my blog...these isoform things, although I know it won't change anything, have me severely confused, and being a Physical Therapist and being this confused about something like this is SO fustrating to me, I keep thinking that understanding these things should be SO easy, it is SO NOT!
I have learned that you are representing ppmd in the upcoming meeting in Uk.I read the agenda and now have a bleak idea of the meeting.My question is that can we hope the exon skipping 45 in near future ,also will the trials be done here in USA.I wish that each exon skipping should be initiated ,regardless of the prevalence of the disease factor as each boys deserves a treatment .
I wanted to drop you a line to let you know that I am still interested in discussing the Quick Trainer Equipment. I realize it's been several months since we've communicated but I was in a serious automobile accident this spring. I have just returned to work and hope to have the opportunity to present to the medical staff the simple mechanism and outstanding benefits of our product.
I just had my second son, conceived through PGD. We did not do an amnio to make sure he didn't have DMD prior to his birth. It was killing my husband after he was born no to make sure so we checked his CPK. It was 129. I was wandering if you knew if the CPK level would be elevated at birth. I thought I remembered a doctor telling me that but I just needed to ease my mind. Thanks so much.
Thank you for your answer. The "it will take a few years" part is very hard to hear... The number of years makes a huge difference.
Don't get me wrong, I would love to see the PPMO approved, the unpublished data shows better efficacy, they are easier to administer (as I understand it the dose required for PPMO is sufficiently low it could be self administered, a lot like insulin. Whereas PMOs need you to sit on a bed with a drip!), good expression in the heart etc. BUT the additional number of years until approval makes all the difference in the world in terms of my son's progression and prognosis.
As for the other things in the pipeline, you and I both know that will take long time until we see one or more of them marketed.
Thank you for your answer. I agree there will be different responses for different mutations; we’ll see that during the clinical trials. What worries me is the choice of chemistry PMO or PPMO. They would need to wait until they obtain some results from the PPMO trial in order to decide correct? That means longer waiting time. There will be million of dollars used in the PMO trials at that point also. What happens is PMO shows benefit but PPMO shows higher efficacy and we already know that it can be delivered to the heart? What happens then with the 15% boys that might be helped by the PMO skipping 51 as opposed to only 5% for PPMO skipping 50?
Thanks for getting back to me. We are 1 1/2 hrs from Phila. I don't want to impose on the wedding time, it's sure to be a busy & happy time. Keep me posted, you can let me know closer to that date. Thx for even considering it:) xoxoxo from Lebanon,PA
Hello Dearest Pat - was truly a blessed honor to meet you at conference. We have a HUGE favor... Our family is having A massive 5K EVENT for our 3 yr old son Levi. It is Oct 3, Sat & we would like to extend you an invitation. I realize this is right after Coach to Cure but we would LOVE to have you there. We would pay for your airfare & you can stay at our home... I undersatnd you may get many of these types of requests but thought we'd try anyway. With great appreciation & love
perlita hains & family
My son Curtis passed away April 18th of this year. we are in the final stages of dealing with his mobility equipment. One key item to be dealt with is his nearly new power chair. this chair is outfitted with a sip and puff component. I would like to be able to link this up with someone in need. Do you have any suggestions? MDC expressed some willingness to assist with transport if it needed to be sent to such an individual.
Thank you, Robyn Hoffman
I was just wondering what the agenda might be for the Round Table meeting they are trying to organize in Sacramento? It is a long drive from Long Beach to Sacramento for a 3 to 4 hour meeting so I wanted to know if it would really be worth the drive.
I know you are an extremely busy person, but I know people listen to you. I just added an open blog post regarding the Help Cure Muscular Dystrophy Phase II project that is VERY close to launch on the World Community Grid.
The project is estimated to run 1 year 9 months (458 centuries of compute time), based upon Phase I participation rates. To cut this to 40 weeks would require 1.2million new members. While this would be a massive challenge at best, all new members contributing to HCMD Phase II will reduce the time it will take to compute this massive project.
I started a Grid team out there named "He's My Son" and have so far gained 44 team members in ~2 months time from family/friends. This team is currently relatively small, but even so are currently contributing 1 CPU year worth of computation every 10-11 days. Interested parties can join WCG and contribute to the Muscular Dystrophy project when it begins without joining this team, but it can be very encouraging to see how the contribution measures up. The link to the team is:
I have long been an advocate for World Community Grid, and even contributed the the Help Cure Muscular Dystrophy Phase I project before I ever had a clue that my son was affected by Duchenne's. I am moreso now and am sending this to you in hopes of gaining a more well known, powerful and trusted voice.