As a mother who lost two sons to this dreadful disease, I am driven to find every way possible to help bring new therapies to families, to end Duchenne. PPMD supports and agrees with all parents urging the agency to accept Sarepta’s submission for the approval of eteplirsen, as well as any other therapy that shows similar promise. The voices of patients and caregivers are critical to the process of drug development and we believe the message is being heard.

PPMD is doing everything it can to enable the FDA to speed the approval of new therapies to all who so desperately need them. We worked tirelessly with others in the rare disease community to pass FDASIA (The Food and Drug Safety and Innovation Act), which included strong language intended to change the paradigm for how the FDA looks at rare disease.

We continue to demand that the FDA use all available tools given to them by Congress to approve rapidly treatments that show safety and effectiveness. This includes eteplirsen and others in late stage development. The moment Sarepta submits an application to the FDA, we will meet with Agency leadership to insist on expedited review and approval.

PPMD remains committed to keep the clinical trial pipeline full, improve care, and bring more potential therapies to fruition—because ALL people living with Duchenne deserve new therapies and comprehensive care.

Last week, Rare Disease Week, over 150 advocates from the Duchenne community, led by PPMD, met with Members of Congress and their staffs to convince them to pass the MD-CARE Act amendments. To date, we have the signatures of over 100 Members of Congress. We cannot forget how we arrived at this brave new era in Duchenne. The MD-CARE Act has paved the way for all treatments, and it is more important than ever to make sure the MD-CARE Act continues to get Congressional support.

Here are three simple actions you can take:

It is too late for my own sons, but it doesn't have to be too late for this generation. Please help us urge Congress to support this critical legislation.

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