PPMD Community

What an odd year it has been. I won’t say it has been a bad year, not entirely, but it has been dark. And I don’t think it’s just me.

Even social media, something I turn to for connection, a familiar friend, a warm embrace…has felt toxic. So many Facebook posts are gas soaked, with every “Like” a potential flame.

This year hasn’t been all doom and gloom though – not by any stretch! There is a light that has shown perhaps brighter than ever before and that light is you, this community. Your strength, resilience, passion, commitment, and courage have been a beacon.

I am hopeful that we are in the midst of a sea change, that the tide is turning for our community. When we look at where we were last Thanksgiving, to where we are today, it’s quite astounding.

Research

PPMD’s Gene Therapy Initiative has been exploring CRISPR/Cas9 as a potential treatment for Duchenne, thanks to your donations last year during the holidays. Dr. Eric Olson and his team at UT Southwestern continue to make progress, as other labs begin their own exploration of CRISPR in Duchenne.

In January, PPMD announced our largest grant award ever when we presented $2.2 million to Nationwide Children’s Hospital to support the microdystrophin gene therapy work of Dr. Jerry Mendell and Dr. Louise Rodino-Klapac. Another example of PPMD’s Gene Therapy Initiative in action, this program continues to quickly move forward with hopes of the first patient dosed before year end!

In February, PPMD announced a $600,000 grant to the New Jersey Institute of Technology (NJIT) and Talem Technologies as part of our ongoing exploration of robotic technology to assist people living with Duchenne. We have always supported innovation, and believe this exoskeleton technology can keep people with Duchenne stronger for longer. What once felt like science fiction is now our reality.

Advocacy

The 21st Century Cures Act was signed into law by President Obama just before leaving office. This legislation contains incentives and innovations that will accelerate therapy development and are designed to translate into therapeutic opportunities for THIS generation living with Duchenne.

Two other critical pieces of legislation—The Better Empowerment Now to Enhance Framework and Improve Treatments (BENEFIT) Act and PDUFA VI—passed the Senate. These policies moved closer to becoming law because of YOU!

You know what didn’t pass? Multiple attempts at healthcare reforms that would have decimated programs our families depend on to offset the extraordinary costs associated with Duchenne. Again, you made that happen. You called, you emailed, you made your voice heard and protected our community.

We rallied together for another Advisory Committee Meeting with the FDA, this time for PTC’s drug ataluren. And while the decision wasn’t the approval we all hoped for, a path forward is still possible with PTC pursuing multiple options while continuing to provide access to the hundreds of families seeing benefit from this therapy.

PPMD continues to help lead efforts to obtain an ICD code for Duchenne and Becker, which we believe will result in easier access and reimbursement to emerging therapies, and we are keeping Newborn Screening a priority so that Duchenne can be diagnosed as early as possible.

Care

Care continues to be a foundation of PPMD’s mission. Over the last year, more clinics joined our prestigious Certified Duchenne Care Center Program, bringing our total to 17 clinics nationwide and plans to expand internationally.

The DuchenneConnect Registry is also going international… now in Australia! Having clinical trials in as many countries as possible helps the whole Duchenne community.

Last spring, PPMD convened the "Unique Burdens of Pediatric Clinical Trials" meeting. As we have watched clinical trials in Duchenne expand, we have witnessed the impact of participating in these trials on children and families. Trials with complex protocols that may extend for lengthy periods of time exert significant pressures that families feel, and exert on each other. We are hopeful that, together, we can help address the burdens being placed on our families involved in these important clinical trials.

Community

Our Every Single One Tour visited seven cities and hundreds of families, connecting families with leading researchers, care providers, and industry partners. 2018 looks equally engaging with eight new locations, in addition to PPMD’s 2018 Annual Conference in Scottsdale, Arizona.

We launched an Online Community Resource Center, the PJ Nicholoff Steroid Protocol was published, Coach To Cure MD turned 10, and the global Duchenne community recognized September as Duchenne Action Month, raising awareness and funds to help end Duchenne.

When I look at this past year (and this was just a taste!), I see courage, commitment, strength – because you never wavered. You stood by one another, and you raised your voice.

Thank you for being my light this year and every year.

Take a moment today and share what your commitment -- to your family, to your loved ones, or to the Duchenne community -- means to you. Click here to share on Facebook.

As always, I am grateful for your support and friendship.

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