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!
- That all boys with Duchenne would have access to optimal care and that we continue to redefine what optimal care means.
- That we understand and address issues related to GERD (reflux or heartburn) in Duchenne and promote proactive care.
- That we develop consensus on treating the hearts of people with Duchenne.
- 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.
- That the community will work with clinicians to develop models for care for adults with Duchenne.
- That we work towards every individual with Duchenne having the opportunity to participate in a clinical trial.
- 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.
- 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.
- 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.
- 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.
- That regulatory agencies listen to the patient voice, learn about benefit/risk in rare disease, and demonstrate flexibility.
- 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.
- That we will work with the NIH's TRND program.
- That we will work with NeuroNext to promote and execute Phase II multi-site trials in Duchenne.
- 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.
- That we work with policy makers and others to develop models that support independence and employment of adults with Duchenne.
- 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.
Pat Furlong, Founding President, CEO
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