Replies to This Discussion

Permalink Reply by christine good on April 6, 2009 at 2:41pm

I am glad that you started this discussion. We go in May for the 1st time. We live on the very rural Oregon coast, so this is a huge trip for us. I have been wondering this since I made the appt. I had been going to doctors for years asking for help because I knew something was wrong. I was told he was autistic, ADHD, spoiled, lazy and referred to parenting classes. At the age of 7, a new doctor ordered his CK's. He is now 8 and we are getting a late start, even though he is still doing very well. Thanks for the reassurance.

Christine

Permalink Reply by Douglas Sanchez on April 6, 2009 at 3:46pm

Michelle,

As a parent who has only just begun to work through the pain, confusion and uncertainty, I believe this is a fully relevant discussion thread. Also, having just returned from first visit to CCHMC I have already begun to answer questions from others on this front as well. Thus I attach here an excerpt, albeit a long one, from a recent e-mail response to a DMD mother:

After absorbing some of this news I asked the neurologist "So where do we go from here? Where do I take him?" and the response was that he was capable of this care. I had already done some research, but not fully since I did not know what I was dealing with. From what I had though I asked further questions about his knowledge of upcoming clinical trials. He assured me he and his partner were MDA doctors (only one other currently listed in Austin area) and that he had the knowledge and connections. He did mention the four "specialty centers" in the US briefly and prednisone/deflazacort as extenders, but the picture painted was nothing but gloom and doom. OK I am not blind to the realities here, but that message does not quite hold true as it was in the past. There are legitimate hopes for us on the horizon and my quick, directed research confirmed that some of what I had seen was indeed promising, even though most was for exon 51 and not for exon 17 where we needed it. The key was in proving the strategies for antisense oligonucleotides and other relevant treatments, and they were indeed moving forward within human trials. He called to discuss carrier testing and I asked him about antisense oligonucleotides and Utrophin efforts and the successes. It was quite clear that he knew what these were, but did not have "latest" data on them and that concerned me. It occurred to me though that as a neurologist he is not deadeye focused on Muscular Dystrophy and that is what I want/need.

I felt that I had to visit Doctor Wong at least one time to determine whether I could REALLY do better than local care. After having been to Cincinnati Childrens Hospital, I fully admit that I do believe I could get all of the tests run through local means. I also feel that I could have them run by pediatric specialists. I am also prone to skepticism and not wasting money. With this in mind I have to say that I felt TOTALLY certain that the visit to CCHMC was the right thing to do and that was the right place to entrust the care of my beloved and only son. As I would expect with any pediatric specialist, these doctors and nurses knew how to work with kids. That is an absolute must! Where I think CCHMC stood out was with their total awareness and knowledge of the DMD/BMD children. It is not just book learning or conference notes they are working from, but real live kids and experience of hands on. While Doctor Wong's team are also officially neurologists, they are not generalized like a local neurologist since they have established their team as MD competency center.

I don't have hard facts here, but rather intuition on what I am about to say, but they are feelings I had and were more or less confirmed by Doctor Wong...

With a mid-sized city you can anticipate that a neurologist may work with "maybe" 3-5 DMD/BMD kids in any given year. As busy as they are, Doctor Wong sees WAY more than that each year. I don't know the numbers, but during our three days at CCHMC I met one other PPMD family directly, saw a couple of kids doing the hallway walking and speed tests, and heard others that I did not see. I also saw ~5 other older kids further along the DMD path. As there are a variety of tests that take you different parts of the hospital, I am sure I probably saw more that I didn't even identify as MD kids. That is a lot of hands on experience. In speaking to cardiologist it was quite clear to me that he worked his specialty, but fully knew how he fit into the total DMD diagnostic package, how the kids were scheduled and when/where he would need to be involved in future visits.

Permalink Reply by Kelvinsmom - Michelle on April 6, 2009 at 5:37pm

I'm glad to see that this could really help folks. I hope so, as just a bit over a year ago, I was searching. I also realize that the discussion forum puts discussions way done the line and putting this information, here in the group, will keep it there for a long, long time for folks to be able to find it. I also realize that there are other places in the US that are great as well, but Cincinnati was where I found myself. I realized that I did forget to mention other items such as Deflazacort that they feel stronger about than some doctors, the human growth hormone that others that go to Dr. Wong are doing great on (if your child falls into a group in which they feel that they need it). The heart (we certainly need to talk about the heart) - Cincinnati Children’s Hospital also has a new member of their neuromuscular team. It is Jeffrey Towbin, MD. Dr. Towbin has been interested in and treating Duchenne patients for over 20 years. His expertise is cardiology. Since the heart becomes "a very big issue" in our sons lives, we certainly want someone who has expertise dealing with it since there are many different approaches that can "help" our sons hearts for some time. When we have been there, Dr. Wong and her staff have many, many rooms full of children waiting to see her, and you think you'll only get a minute with the doctors, but honestly, we have never been rushed and they are always, very thorough, going over every detail, what tests appear needed, etc. They are there to answer every single question you have and they will. I too realize that a lot of folks don't think that any doctor can delay the progression and that it doesn't matter who they go to, but I disagree. There are approaches that prolong many things from happening as fast, like wearing night braces, stretching, massaging etc. to help the heal cords. Boosting some supplements to try to help prevent bone breakage, etc. They all aren't a cure, but going at every aspect of our sons bodies and paying attention to changes through tests, etc. can help find things to help them, well, we hope and pray, until something is there to help them all out for good. We don't know what will happen as far as time of progression, etc., but I'll do anything to keep Kelvin's quality of life as best as it can be in the meanwhile. Michelle

Permalink Reply by Phyllis on April 6, 2009 at 6:57pm

Hi Michelle

THANK YOU so much for posting.. I am very new to this process..Our son was diagnosed in December 08. The parent your referring to in your blog is me... I am absolutely not upset by your post. I am sold I am going to see Dr. Wong in August. feel free to keep this going I have felt so relieved to have an outlet to voice my questions and feelings an for all others out there Doug Sanchez is a wealth of knowledge and has been so helpful to me thanks Doug...Annyone going to Atlanta for the PPMD conference We are tryin to get there and if it happens I would live to put the faces with the names..Thanks to you all ---Phyllis

Permalink Reply by Laurie Paschal on April 6, 2009 at 9:51pm

Hi Phyllis,
We'll be at the conference in June. When in August will you see Dr. Wong. We'll be there the 12th & 13th...plus a few days beforehand.

Laurie

Phyllis said:

Hi Michelle

THANK YOU so much for posting.. I am very new to this process..Our son was diagnosed in December 08. The parent your referring to in your blog is me... I am absolutely not upset by your post. I am sold I am going to see Dr. Wong in August. feel free to keep this going I have felt so relieved to have an outlet to voice my questions and feelings an for all others out there Doug Sanchez is a wealth of knowledge and has been so helpful to me thanks Doug...Annyone going to Atlanta for the PPMD conference We are tryin to get there and if it happens I would live to put the faces with the names..Thanks to you all ---Phyllis

Permalink Reply by Julie Hathaway on April 6, 2009 at 10:23pm

I am in total agreement with Michelle and all that she had to say about Dr. Wong and her team. Brandon is 6 and was diagnosed in October '08. We saw Dr. Wong in December and will see her again in July. We were very impressed with her and her team. They are so compassionate, dedicated, and hands on in every aspect. Dr. Wong has responded to my emails, personally in a very timely manner and is on the cutting edge of trials, studies and the latest research. Her mission in life, I do believe, is to find a cure for this disease. I look forward to meeting some of you at the conference in June. Stay strong in your faith and let's all pray for one another daily. It's what keeps me going!

Permalink Reply by Laurie Paschal on April 7, 2009 at 1:19pm

Ofelia,
Do you happen to know what other centers have been contacted by AVI? I'm really praying there is one in the south somewhere. Would make travel much easier.

Laurie

Permalink Reply by Kelvinsmom - Michelle on April 7, 2009 at 4:24pm

I have only seen Dr. Mendell once in the MDA clinic, he sat down to take all the time in the world that I needed. He and the genetic counselor sent me down to be tested as a carrier that day, and I did, and I was. He certainly is one person in this world who just won't give up until there is help for our boys. He has been working on it since around the time that I was born. We split our visits between Cincinnati and Columbus since we live here in Ohio and both places are on my insurance. I wanted to cover all bases possible, and in my opinion, these two places are amongst the best, if not the best, in the entire US (just an opinion). Cincinnati did take a more progressive approach at first with Kelvin, as he was 5 1/2 when we found out. Either place could certainly end up in trials and it certainly would be great if it was Columbus, as it is closer to home for me. I know Dr. Wong is diving futher into research as well, especially lately. Our next Cincinnati visit is at the end of August, the last week some time. I'll have to look up the dates. Michelle

Permalink Reply by Tamara "Michael's Mulisha" on April 16, 2009 at 12:21am

Just a quick note...we just got back from our first visit with Dr. Wong. It was the best thing we did for our son. We took him to our MDA clinic when he was first diagnosed. We were told, to come back in a year and just let him be a kid. No tests were performed and no discussion of upcoming trials. We left there the same as when we got there. Totally helpless and empty.
Talking with other parents here on this site, we made our appt to go to Ohio. We walked out of there with our heads high filled with so much information on our son's health. Dr. Wong ran tests and went over the results with us immedietly. She spent 2 hours with us just getting every little bit of info on him as she could. Then the next day we spent more time with her going over all the tests. She changed his DX from Duchenne to Becker. (We were never told which one he had). We are going back in 3 weeks for a muscle biopsy. I will only trust her care for my son. She really set Michael aside and examine him as himself. Not as another "DMD case". It was personal and informative. We are now getting night boots and doing proper stretches that no one ever told us to do before. She is all about being pro-active. We like that.
So, thats my 2 cents.

BTW Southwest had some good deals into Columbus then we rented a car for the 2 hour drive. Wasnt bad at all and was well worth it.

Permalink Reply by Jean on April 20, 2009 at 1:01pm

Michelle - Just read your note from April 6, and it was very interesting to me. We have just begun the process of getting a referral to see Dr. Wong fro my two grandsons (7 & 9) who have DMD. No Dr. or clinic near or in Amarillo has the knowledge or facilities that we feel the boys need. Think you did well to post this. Thank you. Jean

Permalink Reply by pam loomis on January 17, 2010 at 9:14pm

I will have to say that Dr Wong is the BEST. She has been our son's doctor since his early teens. Nathan turns 27 on Tuesday, Jan 19th. Our only regret is that we didn't have her in his early years, instead of a doctor that didn't care, since he felt Nathan had a death sentence anyways. Dr. Wong is one of the most caring doctors I have ever met, even making time from her busy schedule for an email from who she calls Nate. She has always seen to it that Nathan had the best care and services provided. We are so fortunate to have her here in Cincinnati- a Blessing!

To all you newbies, I have shared your thoughts and feelings. Get all the information you can at the conferences, hospitals and other groups such as this. You are your child's best advocate.

Looking forward to a cure.

Permalink Reply by Ofelia Marin on April 7, 2010 at 4:55pm

Just a quick note here about Columbus Children's. Dr. Kevin Flanigan (from Utah) joined Dr. Mendell's group and is seeing patients. As you probably know, he is one of the best in the field and was one of the principal investigators for the Ataluren trial among other trials. He is the contact person for Prosensa's upcoming exon skipping trial in non-ambulatory boys starting this year. Dr. Mendell is the contact for AVI's exon skipping trial starting this year. They are also running the losartan vs lisinopril trial among many others. It looks like Ohio is a great place for DMD research and hopefully soon treatment.

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