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Your gift to PPMD is a commitment to accelerating progress

As a pediatric neurologist who takes care of children with neuromuscular diseases, I’ve had the privilege of witnessing first-hand the benefit many of our therapies can have for individuals living with Duchenne. Your belief in PPMD and unwavering support has led to monumental progress for this community, and I am optimistic about what the future looks like from here.

However, a significant gap remains in our understanding of the long-term impact and cumulative effects of multiple therapies on patients after their initial regulatory approval. It is time to establish a new standard of care for Duchenne and Becker muscular dystrophy. Thanks to rapidly evolving technology and patients’ increased ability to share their data, we are now at a critical inflection point and are equipped to address this important question.

PPMD’s Dystrophinopathy Clinical Research Network (DCRN) will allow us to access and aggregate clinical data from multiple sites within the network and develop a better understanding of how effective these FDA approved therapies are for Duchenne patients.

Together, we have an opportunity to revolutionize the management of Duchenne and Becker muscular dystrophy through timely analysis of real world data to inform clinical care in a new era.

This new reality requires a new approach to research. Join me to advance the research evolution. Your gift to PPMD’s DCRN will support groundbreaking collaboration between researchers, clinicians, regulators, advocates and citizen scientists to revolutionize how we study the effectiveness of therapies in the real world.

The progress we’ve made would not be possible without the generous support of individuals and families in the Duchenne and Becker community, along with the visionary leadership of PPMD. I am fortunate to work with a world-class team, including Emma Ciafaloni, MD, Bo Hoon Lee, MD, and physical therapists, genetic counselors, and clinical research coordinators, all of whom are making a real difference for our patients. But our work is far from over.

PPMD’s DCRN will transform how we analyze data and establish new standards of care for Duchenne and Becker muscular dystrophy in a growing landscape of approved and investigational therapies. Give today to play an integral role in the development of the DCRN and its impact on this generation of individuals with Duchenne and Becker.

Warmly,

Sam Mackenzie, MD, PhD

Pediatric Neurologist, University of Rochester Medical Center’

 

P.S. Make your gift now to fund our commitment to evolving research today. Give today.

The post Your gift to PPMD is a commitment to accelerating progress appeared first on Parent Project Muscular Dystrophy.

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