Care Considerations – to be published in Lancet – is available now, online . These Care Considerations set the stage for all boys everywhere to receive optimal care. Families now have access to this document as well as an abbreviated, family-friendly version currently in development. This is your guide and your doctor's guide to good care. Your questions, your concerns are addressed. You now have published evidence about what is best for your son. You no longer have to fight the good fight to obtain good care. Today, it’s here and while some of you may believe in a more aggressive approach, this document reflects what ALL experts agree upon. It is a consensus document.

Victor Dubowitz is famous for saying ‘boys are not mice’ and what happens in the mouse may not exactly translate. In other words, we (researchers) can cure mice, but the task becomes more difficult as you move up the ladder – rodent –dog –monkey – human.

And who hasn’t had millions of Google Alerts about mice? And afterward conversations with a researcher who disagrees with the information. Frustrating! And why? Because animal testing is not standardized.. until now. In mice clinical trials, they had no standard of care! Meetings were recently held to develop SOP (standard operating procedures) for research/animal testing. Otherwise we are not comparing apples to apples.

It’s the same for our sons. Care varies across the US and across the world. It is important to ‘level the field’, to ensure all boys have access to standardized care, which is necessary to improve outcomes today for clinical trials tomorrow.

When Chris and Patrick were here, state of the art care consisted of 5 words “no hope and no help.” Boys with Duchenne stopped walking at 8 or 9. Physicians did not perform baseline studies and no one mentioned bone density. Night splints (AFO’s) and stretching were not recommended. Muscle biopsies were done to confirm diagnosis (the absence of dystrophin). Genetic testing was not on the map. And families were alone…there was no internet.

Today, thanks to your advocacy efforts, we now have Care Considerations, a document of expert consensus about Care – what it should look like, how often to review, what tests, what medicines to add and when. It discusses the need for multidiscipline care (coordinated care that involves many subspecialists), the need for genetic testing and participation in registries. It lays down a basis for good care. This in turn prepares our sons for clinical studies and for researchers, clinicians, and industry to appropriately determine benefit and get therapies approved.

Improved care translates into improved quality of life and extended life span. It provides all of us – researchers and clinicians a level of confidence about care. It also highlights gaps – things we need to think about and improve our base of knowledge.

Print a copy of this document, keep it handy. Use it for reference, for emergencies. Use it for information, for evidence. You now have the back-up you need to ensure your son receives the best care possible and (cross your fingers and pray) the opportunity to participate in promising clinical trials.

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Comment by Julie Garcia on December 27, 2009 at 5:08am
Thank you
Comment by Ivy Scherbarth on December 9, 2009 at 5:14pm
This is fantastic. Period. I love that even in a rigorous, scientific writing style, the tone was positive, hopeful and helpful, paying attention not just to the patient as a person, but to the whole family affected by Duchenne. The recommendations are clear and the consensus obviously based upon experience and common sense. This is exactly the kind of research and reporting that I am so proud PPMD supports and facilitates. I am sending a copy to all of our doctors, therapists and other care practitioners along with their Christmas cards. May each and every family facing DMD receive the kind of care that this document promotes!
Comment by Ian Anthony Griffiths on December 8, 2009 at 5:32pm
Thank you Pat for your kind words. I'm really hoping things like this become rarer now. Its like turning a juggernaut must be even tougher in the states.

Thanks Veronica too.

hugs
Comment by Veronica E. on December 7, 2009 at 9:30pm
I started reading this at work today. I'm SO happy to share it with Max's pediatrician and use it as our own guide in the years to come. I'm thankful it finally exists!

So sorry, Ian, that you've missed out on the care you deserve. Thanks for posting here to give your perspective.

Staff
Comment by Pat Furlong on December 7, 2009 at 8:03pm
Ian,
Your experience is gutwrenching and happens over and over and over again. You are right, we will win and it is because of your courage and perseverence.
Comment by Ian Anthony Griffiths on December 7, 2009 at 4:38pm
Its a great document and I have read it all. The sad thing for me is that I've missed out on this care for so long, my drs have waited too long for me, I had the vent and trach after a period of non monitoring and suffering from respiratory distress and pnuemonia. My cardiologist told me that an ECG showed damage in 2001 but not until 2007 did I receive the heart drugs. It's about time we had this document but its nigh on impossible to get our local health board and UK government to listen even when I shove this in their face.

For me the fight goes on, I can't bear to see another boy struggling with DMD, ill keep on. My dr's are giving me the choice of Palliative care and writing a will, so galling for a 25 year old to face.

The tide is turning and soon we'll win!

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