Today, PPMD joined the Best Day Ever Foundation, CureDuchenne, the Jett Foundation, the Little Hercules Foundation, The Muscular Dystrophy Family Foundation, and Team Joseph in sending a letter to the Federal Drug Administration (FDA) regarding the ongoing review of EMBARK trial data for ELEVIDYS. The letter acknowledged the difficulty in measuring and evaluating Duchenne disease progression in a relatively short study period, but also urged a timely and transparent review of the data.

We recognize and value how the FDA has incorporated the patient perspectives throughout this decision-making process and encourage continued engagement as the review moves forward in an expedited and inclusive manner.

You can read the full text of the letter here.