"And here is more, which I certainly don't fully understand, but I do think I learned something about how the body responds to the treatment. This statement seems to say that the effects on fibrosis are the side-effect of a side-effect…"
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David
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Do we need advocacy efforts around the coverage of exon-skipping treatments by insurance plans?
Started this discussion. Last reply by Keith Van Houten May 18. 4 Replies 0 Favorites
My best research efforts suggest that the cost of AVI or GSK exon-skipping treatments, once approved, would run between 100k - 200k per year, per patient.Further, comments by some folks I know in the…Continue
Exon skipping and young men with DMD
Started this discussion. Last reply by David May 13. 14 Replies 0 Favorites
PPMD has recently posted PDFs from the conference presentations recently in San Diego. Link is at the bottom of this message.In the exon-skipping presentation, on page 23, there was a statment about…Continue
How can parents help along FAST ACT 2012? a.k.a House Resolution 4132
Started this discussion. Last reply by David Mar 22. 1 Reply 0 Favorites
http://www.rarediseases.org/news-events/news/fast-act-2012“Accelerated Approval” refers to a pathway at FDA that allows for…Continue
Summit SMT C1100 utrophin upreg phase 1 trial could start in March, results as early as September 2012
Started this discussion. Last reply by PPMD Feb 16. 1 Reply 0 Favorites
Assuming phase 1 is a success, how can parents help encourage Summit to consider US sites for phase 2? ******** PRESS RELEASE ***************Today's press release announced that the manufacture of a…Continue
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David replied to Amrit's discussion Halo"Boy, even the articles referenced on the Halo web site seem to say very little about how it works. Maybe the mechanism is just beyond laypeople? I dunno.
One abstract did have something interesting, but I sure can't translate it. Maybe somebody…"
David replied to Amrit's discussion Halo"It seems they have raised enough to start phase 2, per this article.
http://www.xconomy.com/boston/2012/05/24/halo-gathers-1-1m-from-patient-groups-for-muscular-dystrophy-drug/2/
For now, Halo has plenty of resources to take HT-100 into the next…"
David replied to Tang Thao's discussion weight control"If your son is on prednisone, you may want to consider switching to deflazacort.
Also, if it is any consolation, my son went through a weight-gain phase at the same age, and it tapered off by age 13.
-David"
David replied to Amrit's discussion Halo"I wait with great hope. I want to stay positive, but the statement about starting the trial "in the second half of 2012" is a bit unnerving. Sounds like another typical biotech press release.
I wish I could tell my customers…"
Keith Van Houten replied to David's discussion Do we need advocacy efforts around the coverage of exon-skipping treatments by insurance plans?"Also, if you get a chance to ask someone in the industry again i'd really be interested in how they'd respond to my question..."
Keith Van Houten replied to David's discussion Do we need advocacy efforts around the coverage of exon-skipping treatments by insurance plans?"David, the most effective thing you can do is to show up at a local event your congressman is holding, introduce him to your son and have him understand how these policy issues impact real people. "
David replied to Tulika's discussion Worlds first approved stem cell treatment"Ah...ok I get it. Or more to the point, I have some weekend reading to learn what EAP is and how it works."
David replied to David's discussion Do we need advocacy efforts around the coverage of exon-skipping treatments by insurance plans?"I don't pretend to understand how insurance companies make these decisions, much less what the law says they must do. I've tried directly asking my provider (Blue Cross of Oregon) but they won't discuss hypothetical situations like…"
Keith Van Houten replied to David's discussion Do we need advocacy efforts around the coverage of exon-skipping treatments by insurance plans?"Out of curiosity, under what basis would an insurance company be able to deny coverage for an FDA approved treatment, for a disorder which has no other options?
Even if the Affordable Care Act is struck down? The Act is what's…"
David replied to Tulika's discussion Worlds first approved stem cell treatment"The article is confusing.
It states "First approved treament" and yet it also states "Prochymal is currently available in several countries, including the United States, under an Expanded Access Program"
If it is…"
David posted a discussionDo we need advocacy efforts around the coverage of exon-skipping treatments by insurance plans?
My best research efforts suggest that the cost of AVI or GSK exon-skipping treatments, once approved, would run between 100k - 200k per year, per patient.Further, comments by some folks I know in the health insurance biz indicate that these treatments would likely NOT be covered by private insurance, even if the Affordable Health Care law is fully or partially struck down by the Supreme Court this summer. I don't pretend to understand the legal issues here, so hopefully this is simply wrong.Do…See More
David replied to wainaina muiruri's discussion Proteins"Our clinic has a great nutrionist who we've met with several times regarding my son's diet. She has never said anything about limiting proteins."
David replied to christopher lundy's discussion how can i help"I would seriously consider purchasing a scooter for you son, not for regular use, but for use in these sorts of events.
Other kids LOVE the machine, are envious even. It will allow him to participate at "regular" speed. It can be very…"
David replied to Marks33's discussion COQ10 pilot trial"Mark,
It's a bit rambling, but check out this discussion which includes many parents and many different recommendations. My son is 14, takes 900mg/day of Idebenone with now discernible…"
David replied to Peter's discussion Ataluren can be bought NOT FOR HUMAN OR VETERINARY DIAGNOSTIC OR THERAPEUTIC USE."I've seen similar scams about ACE-031 available for purchase from various muscle / body building web sites. They are preying on our emotions to expand their markets. You'll be lucky if it is only a sugar pill. "
Profile Information
- About me:
- Father of a DMD boy
- About my family:
- Son, 11 Joe
Daughter, 9, Maddie
- Name(s) of child(ren)/individual(s) with Duchenne:
- Joe
- Age(s) of child(ren)/individual(s) with Duchenne:
- 13-18
- City and State:
- Sacramento CA
- Country:
- USA
Comment Wall (11 comments)
- At 9:23pm on September 3, 2011,
Joshua's mom said… Hi David,
You probably never expected the world to be this small!! I'm so glad you talked to him. He's one of the best advocate doctors you will know. You are more than welcome. We are all DMD family.
- At 11:39pm on August 23, 2011, Joshua's mom said…
- A few years ago he came here to Canada. He then went back to Sacramento in October of last year. I did find a link for him in San Diego. Dr. Skalsky No doctor we've met is as optimistic as he was.
- At 8:19pm on August 21, 2011, Joshua's mom said…
- Here is the link to Dr. Skalsky. Everyone here, in Manitoba, virtually cried to see him go. He was the first on who actually knew the deletions & duplications the kids had before we met him. He knew more about DMD than the geneticist did! He was also the one who pushed for Idebenone here in Canada. That was the main reason why he came here. Most all DMD kids are now on Idebenone because of him!!
- At 1:40pm on August 21, 2011, Joshua's mom said…
Hi David, I saw your post on Idebenone regarding your son's doctor suggesting it. I also see you are close to Sacramento. The doctor we had here who suggested Idebenone has moved back to Sacramento. His name is Dr. Andrew Skalski. I just had to ask if you are familiar with him or his team?
Naomi
- At 10:58pm on January 18, 2011,
Nguyen Van Anh said… Hi David,
Oh, really, I didn't remember. I'm sorry for my mistake.
And I'm very happy to be your friend. .
But there're one thing, I want to ask you why your son need exon 17,18 skipping? I think it should be exon skipping 17 only.
Thanks
Van Anh
- At 9:20pm on January 12, 2011, Nguyen Van Anh said…
Hi David,
My son needs exon 17 skipping (he has delection of exon 18-25), and in your comments left on delection group , you mentioned about Katerina with exon 17 skipping. Please send me her contact. I really want to find someone with my boat.
Thanks
Van Anh
- At 1:35pm on November 29, 2010,
cheryl cliff said… - David,
Please contact me at cherylcliff@gmail.com. I have some information you might find helpful regarding your seeking an IND.
- At 3:48am on July 8, 2010,
RAKTIM SINGH said… - Thanks very much David for the information on ACE-031.I am praying for the success for ACE-031 as that will help all DMD kids.
Thanks once again.
MAY GOD BLESS YOUR FAMILY.
Raktim
- At 9:34am on July 7, 2010, RAKTIM SINGH said…
- Hi David,
Do you any information on ACE-031. Personally I am quite excited about that. This surely can give our kids some more strength, muscles and time(especially in the light of latest delay in exon trials in US).
I am aware of ACE-031's canada's PH2 trials. Are there any planned in US also. Any other info related to this will surely help.
Thanks
MAY GOD BLESS YOUR FAMILY.
Raktim
- At 10:06am on June 30, 2010, RAKTIM SINGH said…
- Thanks David for the information. So as I understand, cocktail of medicine which can skip more than 4 exons can be less efficient (but hopefully 2 exon skipping cocktail can still work.)
Thanks once again.
Raktim
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