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January 17, 2007
Parents Know
Once upon a time there were a group of parents whose children were diagnosed with Duchenne. They felt alone and isolated. Little was known about the disorder, and parents were instructed to watch and wait. It was heartbreaking and impossible.
Clinical trials were conducted throughout the 80’s, and the dystrophin gene was identified in 1986. Still, almost nothing was known about the disorder’s pathology. Investment in Duchenne research was scarce. Parents wanted and needed more.
In 1994, a small group of parents decided that it was time. Time to organize. Time for researchers to speak with clinicians. Time for parents to connect with experts. Time was precious. Parents led the charge and PPMD was born.
A real investment was made in our sons’ futures. Research centers. Lobbying efforts. Parents went to Washington to raise awareness, and Congress listened. The MD CARE Act. NIH funding.
Meanwhile, the field of DMD research matured. The idea of developing treatments seemed possible. Parents were recognized as partners. After all, parents know what’s at stake and are willing to do anything – go anywhere – for progress.
Parents considered the ‘no hope and no help’ approach unforgivable. Attitudes changed. The DMD world changed. The entire community felt different. Our sons were expected to grow up. There was hope. And parents found that hope was contagious!
The diagnosis remains a crushing blow. But there is anticipation. And when change is tangible, parents want – and expect – that change to happen as quickly as possible.
A typical PPMD board meeting discussion centers on research. But in early 2006, the board began to ask specific questions. Why was there fragmentation in the DMD community? Why were there roadblocks in treatment development? The PPMD board – mostly parents – agreed to champion efforts to create a global collaboration.
We were all organizations capable of reaching large number of DMD families around the world. Could we collaborate to identify issues critical to the development and dissemination of effective treatments? Could we work together at all?
Indeed, how could we refuse? DMD boys – our boys (and some girls) – needed us to work together. To advance treatments, nothing short of global cooperation was acceptable.
Meetings were scheduled. We agreed that the field was ripe with promise. The timing was right.
This weekend, PPMD will sign a Memorandum of Understanding with AFM, MDA and UPPMD (PPMD global affiliates). We will work together to accelerate the development of treatments. This will include a global registry of patients, an international clinical trial network, a research database outlining all projects funded, and the development of Standards of Care.
We will continue to break down barriers. Reduce fragmentation in the community. Once again, parents led the charge.
And thank goodness. Parents, grandparents, sisters, brothers, uncles, aunts, friends. We know what’s at stake.
Happy New Year!
Posted by ppmd at 12:49 PM | Comments (0)