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.