I wanted to share with you a few of my resolutions for 2012...mainly goals for PPMD and the Duchenne community, but also a couple of personal reflections. 2011 was an incredible year for Duchenne. We made great strides in research, care, advocacy, and in raising awareness. But there is still so much I want to tackle...we all want to tackle.
 

Read over my resolutions and then please take a moment to share some of your own. This time next year, we can all check back and see how we did!
 

  1. That all boys with Duchenne would have access to optimal care and that we continue to redefine what optimal care means.
  2. That we understand and address issues related to GERD (reflux or heartburn) in Duchenne and promote proactive care.
  3. That we develop consensus on treating the hearts of people with Duchenne.
  4. That we promote and use devices to improve how the heart works, like implantable cardioverter defibrillators and left ventricular assist devices, and cardiac transplantation when appropriate.
  5. That the community will work with clinicians to develop models for care for adults with Duchenne.
  6. That we work towards every individual with Duchenne having the opportunity to participate in a clinical trial.
  7. That we will use even more effectively our ever growing number of animal models (zebra fish, mouse, dog, pig) will continue to provide relevant/reliable data for decision making about which drugs have the most promise.
  8. That drugs that are already being used for other easons, and that might also beused to slow progression in Duchenne, will be in clinical trials.
  9. That all of the community's efforts are able to accelerate on the development and regulatory path, including:
    • Potential steroid replacement drugs (Delta 9,11 compound, VBP15 (ReveraGen) and P38 inhibitor (Pfizer)
    • nNos –Sildenafil and Tadalafil
    • anti-fibrotics – Halofuginone (Halo)
    • Myostatin inhibition – ACE-031 (Acceleron/Shire) (PTC Therapeutics) (Pfizer)
    • Idebenone (Santhera)
    • Antisense oligonucleotides (Prosensa/GSK and AVI)
    • Ataluren (PTC Therapeutics)
    • Utrophin up-regulation – Biglycan (Tivorsan), SMT C1100 (Summit). Catalyst (PTC Therapeutics), Tejvir Khurana's compounds at U Penn.
    • Gene Therapy – U7 (L. Garcia), follistatin (Mendell), mini/micro dystrophin (Mendell), utrophin (Chamberlain), micro dystrophin (Chamberlain)
    • Check out PPMD's website for a comprehensive therapeutic pipeline.
  10. That we have opportunities to explore compound libraries (which are huge collections of potential drugs that are waiting to be checked out) of willing pharmaceutical companies to discover potential therapeutics for Duchenne.
  11. That regulatory agencies listen to the patient voice, learn about benefit/risk in rare disease, and demonstrate flexibility.
  12. That different and better ways of looking at Duchenne progression and change, such as MRI (ImageDMD) and others will be allowed for use as endpoints in clinical trials.
  13. That we will work with the NIH's TRND program.
  14. That we will work with NeuroNext to promote and execute Phase II multi-site trials in Duchenne.
  15. That we are able to work with payers (insurance) to ensure all individuals with Duchenne will have access to standards of care treatments, and even cutting-edge treatments.
  16. That we work with policy makers and others to develop models that support independence and employment of adults with Duchenne.
  17. That in 2012, SAFE and EFFECTIVE therapies will become approved for Duchenne.

 

And on a personal note:

  • I’ve started taking bikram yoga and even after a near-death experience in the 105 degree heat, am determined to continue.
  • I will drink more water and less Diet Coke.
  • I will be the best mom for my daughters and the best daughter for my aging mother.


Thanks for reading, and please share your resolutions.

Happy 2012!

 

Pat Furlong, Founding President, CEO
Read more PPMD Staff Blogs

Views: 822

Comment

You need to be a member of PPMD Community to add comments!

Join PPMD Community

Comment by Liisa Underwood on January 5, 2012 at 9:42am

Michelle, I totally understand.  I'm sorry!

Comment by Michelle Scaglione on January 5, 2012 at 2:10am

  Lisa

Johnny has been on Nexium for about 5 years.  Without the Nexium he is in extreme pain from heartburn.  I had to give him alot of Mylanta before the Nexium and it didnt work.  I dont want to try other meds on Johnny and watch him needlessly suffer because the insurance has decided not to pay for the Nexium.  They dont care what he takes as long as it isnt expensive. 

Pat, I would love to start advocating but to be honest I tried fighting with the medical for 2 weeks with his DRS help and only got so aggravated I thought I would have to be sedated!  I dont have have the strength mentally or physically at this point.  I choose to take care of Johnny full time and that leaves me no time for anything else.  I will just pay the $200.

Comment by Liisa Underwood on January 4, 2012 at 1:32pm

Our oldest son who has GERD and not DMD was recently taken off Nexium. In my non-medical opinion, I would NOT suggest Nexium for our boys with DMD, especially if they are on a steriod. I was told that Nexium is a Calcium inhibitor which is an unnecessary side effect for our boys. Michelle, has there been an issue with using other acid inhibitors? 

FYI, before the insurance would approve to pay for Nexium, my son had to prove the other medications did not work for him. They never did, so he was approved and the Nexium worked great. It turns out though that the fault was not the other medications.  They should have worked for him. After his second endoscopy, he finally showed signs of gluten intolerance.  He tested negative for Celiac, but positive for needing a gluten-free diet.  Once his reflux issues improved with the new diet, we weaned him off the Nexium and he is back on Pepcid.

Also, be careful with Zantec.  Both my boys and a friend's kids all had major behavior changes with Zantec.  It's not a listed side effect but my friend (a doctor) and I are 100% convinced the change in behavior is due to the medicine.

My 10 cents for what it is worth since I'm not a medical professional.


Staff
Comment by Pat Furlong on January 4, 2012 at 12:24pm
Michelle,
Here is what I understand and perhaps others have additional information. Insurers (payers) agree to cover drugs based on a compendium. The compendium is a list of drugs and indications for the use do those drugs. There are several of these and therefore some variability, based on the relationship with the payer,
Physicians with expertise in Duchenne recognize and treat symptoms of GERD, though there have been no studies, trails and few case reports. We are working on a meeting fouled in these issue, but I realize this won't solve the problem you have. I would suggest trying:
1. Ask the physician to write a letter to describe your sons GERD and the Ned for Nexium
2. Is the physician using the ICD 9 code for GERD in your sons diagnostic criteria?
3. With this in hand, call CMS and start advocating.

I will ask a few of the doctors for additional suggestions. Clearly, this is something we have to address not only with Nexium but others proved drugs that are or will be used for individuals with Duchenne.
Comment by Michelle Scaglione on January 4, 2012 at 12:51am

Pat

In your blog you mentioned GERD.  Is there any data to prove to the insurance that men with Duchenne and who are on steroids need medications like Nexium?  My sons insurance will not approve it and it costs $200 dollars a month.

Need help using this community site? Visit Ning's Help Page.

Members

Events

© 2019   Created by PPMD.   Powered by

Badges  |  Report an Issue  |  Privacy Policy  |  Terms of Service