Hope Through Research

At MDA, we take a big picture perspective across the full spectrum of neuromuscular diseases to uncover scientific and medical breakthroughs that accelerate treatments and cures. The power in our research approach is that we can often apply learnings from one disease to progress in others to bring urgently-needed answers to our families.

Formulation: Getting All the Right Ingredients

This step goes along with delivery and dosing, and refers to how to make the final drug. Any drug that you take includes ingredients other than the active drug itself — packing components that turn it into a pill, liquids to turn it into an intravenous fluid, and possibly other components that help the drug get to where it needs to go or disperse it through the tissues.

Developing a safe formulation that helps the drug get to the right place can be critical in making the drug actually have an effect in people.

For example, the utrophin upregulation drug tested by Summit PLC and BioMarin showed no effect in phase 1 trials, but Summit still believes that the active ingredient works. Unfortunately, an ineffective formulation meant that not enough drug ever reached the muscle to have any chance of working. The company has developed a better formulation, which appears to get to higher levels in muscle tissue.

At the end of the formulation stage, the developer has a drug that can be shipped to a clinical trial site and administered.

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