We’re so close to our goal to fund CRISPR/Cas9!…

Your donation will be DOUBLED. Now is the moment to give.…

Help us raise $250,000 by 12/31!…

We have all had frustrations getting healthcare paid for, whether it is getting access and coverage for appointments, procedures, equipment, and/or medications.

Coverage is especially difficult when new medicines or procedures are recommended. Parents, patients, and…

Help us raise $250,000 to fund CRISPR/Cas9.

After a few months of relative quiet, noise has returned to the Killian house in full force! For the holidays, all five kids are back under one roof with a revolving door of friends and endless groceries. Even with all the chaos that entails, this is my favorite time of…

When PPMD convened the Contemporary Issues in Duchenne Cardiology meeting with National Heart, Lung, and Blood Institute (NHLBI) in July 2014, there were several recommendations.

The strongest recommendations included the use of cardiac…

It is the holidays – time for giving and laughter and hope. Hope comes in many forms, but I would imagine that for many reading this blog, hope comes in the form of a cure for Duchenne.  

Could CRISPR/Cas9 offer hope for treating Duchenne? 

As background, CRISPR/Cas9 is a…

When I joined PPMD in 2012, care for Duchenne was all over the map. 

Sadly, there were over 150 clinics spread across the US, but there was no oversight of these clinics – the community did not know who was providing care, the level of care being provided or whether anyone at the…

The Duchenne Community’s Voice Was Essential Every Step of the Way & Contains PPMD’s Provision to Strengthen Patient Focused Drug Development Efforts

To everyone in our PPMD community who called, reached out, emailed, and met with your elected officials –…

Duke University marked our final stop on PPMD’s 2016 Every Single One Tour, held on November 2. We were thrilled to be holding another tour stop at one of…

We need you to call your two US Senators TODAY with one simple message – VOTE YES ON 21st CENTURY CURES!!

With just days remaining in the current Congress, this bill represents the extraordinary efforts of our PPMD community’s work and outreach with…

We have so much momentum. Let’s seize this moment.

First of all, let me say THANK YOU! Your gift to PPMD on #GivingTuesday helped us surpass our goal, and we could not be more grateful. It is a wonderful way to kick off our holiday season—and we wanted to make sure you were among the…