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Basic Research

This is the “how does that work?” stage: it’s figuring out how normal muscles operate, understanding what mutations cause disease, and how those mutations interfere with normal muscle function.

Representing years of investigation, the results are typically layered on top of one another and collectively build-up a detailed picture of biological processes. This kind of science is almost always “hypothesis-driven” using the scientific method that you learned about in school: You have an idea, you think of ways to test it (ideally giving you a clear yes or no answer), and you really want other people to be able to test the idea in different ways and still get the same answer.

Back to the Drug Development Process

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There has never been so much momentum in the Duchenne community. Help us keep moving forward by donating to fund CRISPR/Cas9 gene‑editing technology. Every gift to PPMD is being doubled until we raise $250,000—but we have to reach this goal by 12/31!