PPMD Community

Dear Friends,

 

It has been an incredibly intense few months as we hit the homestretch of 2015 and head into a promising 2016. One Advisory Committee meeting is down and we have another quickly approaching most likely in late January 2016. BioMarin, Sarepta, and PTC are all expected to find out if their therapies have been approved in the coming months.

 

The stress of this process has weighed heavily on the entire community. Even if you or your child would not directly benefit from drisapersen, eteplirsen, or ataluren, I’m sure you understand how these decisions will affect therapies waiting in the wings that have the potential to benefit every single family in our community.

 

There has been more to this year than just Ad Comms though. For the first time in rare disease history, a patient community submitted a proposed draft guidance to the FDA to help educate them on a disorder. The guidance was accepted in 2014 and this past summer the FDA released their own version of a Duchenne guidance. Now we can get an inside look into what the agency is considering and weighing as priorities when they consider potential therapies in Duchenne. 

 

Our community spoke, different voices unified in mission, and we were heard.

  

PPMD_BD_Email_TGIVS_CenterCallOut.jpg

 

And we continue to share our stories through benefit-risk reports and patient-preference studies. We are not only changing the landscape of Duchenne, we are affecting positive change in the broader rare disease community.

 

But there is too much work left to be done to ever rest on our laurels.

 

PPMD has a history of funding early stage research, supporting innovative ideas and helping them get to the next step on their long journey to eventual approval. This year we not only supported drugs that are approaching the finish line, but also several therapies just out of the gate, and we are excited about new research projects in 2016.

 

We continue to seek legislative solutions to address critical issues like access and reimbursement, and we insist that the patient voice be inserted into the decision-making process. Through our clinic certification program and our ongoing webinars and roundtables aimed at addressing each important body system, we continue to emphasize the importance of optimal care and the need to continually improve and update our approach.

 

In a disease full of such uncertainty, one thing we do know is that better care translates into a better quality of life.

 

This fall we announced Phase II of Decode Duchenne, again convening important members of the community by welcoming support from BioMarin, PTC, and Sarepta. Decode Duchenne, powered by the DuchenneConnect registry, helps families with a Duchenne diagnosis receive free genetic testing and counseling.

 

In the New Year, we will continue to expand the outreach of Decode Duchenne so that all families living with a Duchenne diagnosis are armed with the genetic information they need to receive appropriate care and access relevant clinical trials. And we – the international Duchenne community – will gather in Orlando next June for our Annual Connect Conference!

 

There is so much happening – and it’s overwhelming, and exhausting, and exhilarating, and heartbreaking, all at once.

 

On Thanksgiving Day though, I urge you to take a moment and find time to be thankful.

 

Maybe it sounds cliché. I just think that in today’s world with all the noise, all the intensity, it’s important to take a deep breath and just focus on what there is to be grateful for.

 

This time last year, I wrote to you excited to announce that I was becoming a grandmother. Now I have a one year old in my life and it’s hard to remember a time without her. This new miracle, this new energy in my family has been one of the greatest blessings for us in a long time. Watching Camille discover the world and navigate the world leaves me in awe. Her determination, tenacity, adaptability, curiosity, and passion for life reminds me so much of this community. This year, I am so grateful for this beautiful grandchild.

 

And I’m so grateful for you.

 

PPMD believes in the strength of this community. We are an incredible family if you will, sharing a journey none of us expected to be on. And every person affected by Duchenne deserves treatments, options, and the opportunity to fulfill all the potential he or she has been given. Today, PPMD is thankful for you and your family, and we promise to continue to do everything possible until we end Duchenne for every single one of you.

 

Happy Thanksgiving and thank you for being our family.

Best wishes,

 

ppmd pat signature.jpg

 

Pat Furlong

Views: 399

Comment

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

Join PPMD Community


Staff
Comment by Pat Furlong on November 26, 2015 at 10:41am

David, I can imagine your heart is breaking.   I wish, for all of us, we soon have novel trial designs that are inclusive of everyone, walking or not, approvals of new medicines and robotics that compensate for muscle loss.  You are in my thoughts today and everyday.  

Comment by David on November 26, 2015 at 10:32am
Im thankful for PPMD. My son will spend his first Thanksgiving without being able to feed himself. We have no trial opportunity in near term. You are all we have left.

© 2017   Created by PPMD.   Powered by

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