For Researchers & Industry
Research is crucial to PPMD’s goal of ending Duchenne and PPMD has been at the forefront of every breakthrough in research in Duchenne and touched every clinical program in some way. We support research through promoting collaboration among scientists; spearheading public-private partnerships that catalyze discovery, development, and clinical research of the most promising compounds; and funding research that will move the field forward. In research, as in all things that PPMD engages in, people with Duchenne come first and we measure what we fund against what it will provide to our community. Our most recent investments into gene therapy demonstrate our commitment to research that will provide meaningful benefit to our community.
As the Duchenne research landscape has broadened dramatically since PPMD was founded in 1994, so has our research strategy. In the early years, PPMD funded two to three projects a year, mainly in exploratory research and drug development. We were still learning about targets and how to identify lead candidates to put through the rigorous testing that leads to human clinical trials, but our the portfolio of projects we invest in has expanded enormously.
Today, we have a varied portfolio of early stage projects, similar to those we funded a decade ago, as well as our ongoing support of clinical trials, tools to accelerate clinical trials, technologic and assistive devices, and meetings that bring researchers, drug developers, regulators, funders, and patients together to find a cure for this generation of individuals living with Duchenne.
PPMD is the only muscular dystrophy research organization:
- To invest in a broad research portfolio.
- To explore short-, mid-, and long-term treatments and therapies.
- To leverage $200 million into research centers and $22 million into Duchenne-specific programs at the Centers for Disease Control.
- To leverage $15.4 million from the National Institute of Health (NIH) into PPMD’s drug discovery program.
In early 2008, PPMD launched The Duchenne Registry (formerly DuchenneConnect), which brings together all members of the Duchenne community: patients, families, and medical and research professionals.
In this section